Wolters Kluwer Tax & Accounting explores expansion of the definition of qualified medical expenses

Wolters Kluwer Tax & Accounting:

What: In recent years when presented with the opportunity, the Internal Revenue Service (IRS) has identified a number of expenses as qualifying medical expenses for purposes of the itemized deduction for medical expenses or for qualified distributions from health savings accounts or flexible spending accounts. These have included smoking cessation programs, weight loss programs, and gluten-free products for celiac disease. Now, in a private letter ruling, the IRS has spelled out the circumstances under which genetic testing might qualify as a medical expense.

Why: While private letter rulings cannot be relied upon by taxpayers other than the taxpayer to whom it was issued, the ruling on genetic testing does indicate the IRS thinking on the matter and how they might treat similar situations:

Who: Federal tax expert Mark Luscombe, JD, LL.M, CPA, Principal Federal Tax Analyst at Wolters Kluwer Tax & Accounting, is available to discuss these developments with respect to genetic testing and qualified medical expenses in general.

PLEASE NOTE: The content of this article is designed to provide accurate and authoritative information in regard to the subject matter covered. The information is provided with the understanding that Wolters Kluwer Tax & Accounting is not engaged in rendering legal, accounting, or other professional services.

Contact: To arrange interviews with Mark Luscombe, other federal and state tax experts from Wolters Kluwer Tax & Accounting on this or any other tax-related topic, please contact:

MARISA WESTCOTT 212-771-0853 marisa.westcott@wolterskluwer.com

View source version on businesswire.com: https://www.businesswire.com/news/home/20190919005224/en/

More here:
MEDIA ALERT: IRS Approves Medical Expense Deduction for Genetic Testing - Yahoo Finance

Recommendation and review posted by Bethany Smith

Harmfulmutations in the BRCA1 and BRCA2 genes (BRCA1/2) are correlated with increased risk fordeveloping peritoneal, fallopian tube, ovarian, male and female breast,pancreatic, and aggressive prostate cancers. The United States PreventiveServices Task Force (USPSTF) recently updated their recommendations for riskassessment, genetic counseling, and genetic testing for BRCA1/2-relatedcancers from their 2013 recommendation.1

Breastcancer is the most common cancer following nonmelanoma skin cancer in women inthe United States, and it is the second leading cause of cancer death. BRCA1/2mutations occur in approximately 1 in 300 to 500 women, and these mutationsaccount for 5% to 10% of cases of breast cancer and 15% of cases of ovariancancer.1

New Inclusions

In its 2019 recommendation, the USPSTF recommends evaluating women who have a personal or family history of peritoneal, fallopian tube, ovarian, or breast cancer or who have ancestry with harmful mutations in the BRCA1/2 genes with a familial-risk assessment tool. Patients who receive a positive result on the risk-assessment tool should receive genetic counseling and genetic testing if indicated.1

Forwomen without a personal or family history of these cancers or a family historyof harmful BRCA1/2 mutations,the USPSTF recommends against routine risk assessment, genetic counseling, andgenetic testing.1

Nursenavigators are likely to play an important role in facilitating thorough,in-depth conversations with patients about routine assessment, geneticcounseling, and genetic testing. In fact, a 2015 study on efficiency inidentifying cancer patients who should undergo genetic and genomic testing indicatednurse navigators were particularly well positioned in the continuum of cancercare to facilitate timely testing in compliance with recommendations from theNational Comprehensive Cancer Network.2

Support for the Updates

Toupdate their recommendations, the USPSTF evaluated evidence on risk assessment,genetic counseling, and genetic testing for BRCA1/2 mutations in women without symptoms who had never beendiagnosed with a BRCA-related cancer and in women with a prior diagnosisof peritoneal, fallopian tube, ovarian, or breast cancer. Recommendationsindicated a moderate (grade B) benefit to assessment, genetic counseling, andgenetic testing in women with a family or personal history that correlated withincreased risk for peritoneal, fallopian tube, ovarian, or breast cancer or whohave family with harmful BRCA1/2mutations. For women without such personal or family history of cancer or BRCA1/2 mutations, the USPSTF gave a gradeD recommendation, discouraging the service from being used.1

Agrade of B means the USPSTF recommends the service offered as having highcertainty that the net benefit is moderate, or there is moderate certainty thatthe net benefit is moderate to substantial. A grade of D means the USPSTFdiscourages the use of the service as having moderate or high certainty thatthe service has no net benefit or that the harms outweigh the benefits.1

Notably,this updated recommendation now includes women with a previous history ofbreast or ovarian cancer who are considered cancer-free and includes ancestryas a risk factor. A perspective piece contextualizing the USPSTF recommendationalso noted that importantly, but not included in this recommendation, BRCA1/2status is relevant for patients with newly diagnosed early stage breast cancerfor surgical decision making and can also be used to determine appropriatetreatment of certain advanced cancers.3

Althoughthe authors of this perspective emphasized the importance of expanding theUSPSTF recommendation, they also lauded the clear recommendation of identifyingpatients with deleterious BRCA1/2mutations as potentially lifesaving and should be a part of routine medicalcare.3

Originally posted here:
USPSTF Expands Criteria for Recommended BRCA-Associated Genetic Counseling - Oncology Nurse Advisor

Recommendation and review posted by Bethany Smith

Previous studies have shown that the standard test for prostate cancer (prostate-specific antigen or PSA) would not work as a screening tool for the general population as it is not reliable enough.

But the new study found PSA tests were more likely to pick out more serious forms of prostate cancer in men who carry the BRCA2 gene fault than in non-carriers.

This means men with the faulty gene could benefit from regular PSA testing.

The study - published in the journal European Urology - included data for 902 BRCA2 carriers and 497 BRCA2 non-carriers.

All men were offered a yearly PSA test for three years and those with elevated PSA reading were offered a biopsy to confirm whether they had cancer.

The researchers found that men who carry the BRCA2 gene fault were almost twice as likely to be diagnosed with prostate cancer as non-carriers.

Those with the BRCA2 gene fault also had more serious tumours - with 77 per cent of men having clinically significant disease compared with 40 per cent of non-carriers.

Men with the fault were also diagnosed at a younger age - at an average of 61 compared with 64 for non-carriers.

Experts estimate that about one in 300 white men could be carrying the genetic fault, but not all of them will develop prostate cancer.

Study leader Rosalind Eeles, professor of oncogenetics at the Institute of Cancer Research, London, said: "For women who undergo genetic testing, options are available to them if they carry a BRCA fault, including preventative surgery and increased screening.

"But there's no prevention pathway in place if men decide to find out if they're a carrier, which is why our research is so important.

Read more here:
Men with 'Angelina Jolie gene' at double the risk of prostate cancer - Telegraph.co.uk

Recommendation and review posted by Bethany Smith

I have two 23 & Me DNA test kits collecting dust on a shelf in my linen closet. The idea of spitting in a vial and possibly finding out that I have some life-threatening condition lurking in my DNA just waiting to make its fatal appearance freaks me out. I know my thinking is extreme, that there are incredible benefits to knowing your health risks before symptoms show, but I cant control my concerns. Sure, I could opt out of the health results and just learn my ancestry, but I already know my test will come back that I am at least 99 percent Ashkenazi Jewishboth sides of my family emigrated from neighboring towns on the Russia-Poland border. My husbands triplet brothers had the same ancestry results on their DNA tests so there will be no fun surprises for him either. Thats why his test still remains sealed next to mine.

But last month I did end up spitting into a DNA kits vial (they require a ton of spit, by the way!), however it wasnt to learn about my familys history or my health future. I decided to do screening through a saliva sample to determine if I am a genetic carrier for any conditions that I may pass down to my future children.

When my husband and I got married almost a year ago, I knew genetic screening was something we would do ahead of trying to get pregnant. If you are a carrier of a condition and your husband is not, your child is not at risk for the condition. However, if both you and your partner are carriers of the same condition, the odds go up to 25 percent for each pregnancy (are you getting flashbacks of Punnett squares from biology class yet?). Since both my husband and I have Ashkenazi Jewish ancestry, we are two times as likely to be carriers of fatal genetic conditions like Tay-Sachs or Fragile X syndrome. So why wouldnt we do a test that can help us ensure the health of our future children?

As an editor for Parents.com, I know that most children born with genetic conditions have no family history of the disorder. I read and report on parents raising children with life-threatening genetic conditions they were not anticipating during pregnancy. Some know their childs diagnosis at birth and set up GoFundMe pages to tackle the healthcare bills that pile up. Other parents know in their gut that something is wrong, but then it takes years for a doctor to track the genetic mutation causing the issues. I want to give my future children the best opportunity to have a happy, healthy life. If that means I need to spit in a vial to find out their risk for a scary condition and make hard decisions when I get the results, that is exactly what I am going to do.

My first step was to call my OB-GYN to make an appointment for blood work. It turns out that genetic screening before pregnancy is considered optional by many insurance providers, despite my familys ancestry and risk, so testing could cost thousands of dollars out of pocket. No thanks! I started Googling other options. Thats how I came across JScreen, a genetic screening saliva test that screens your risk for more than 200 diseases that are predominant in the Jewish community. The not-for-profit program is based out of Emory University and tests samples in a CLIA-certified lab (so I knew it was legit) and is funded by several Jewish organizations so each kit only costs $149 whether your insurance covers testing or not. To order a test, I had to provide my doctors information so she can sign off and receive the results (they are shared through a HIPPA-compliant database), and thats it. While you dont have to be Jewish to order a JScreen kit, there are other at-home genetic screening kits available on the market, including Invitae, which is ordered through a similar process with your doctor and costs $250. For me, JScreen made the most sense.

Taking the JScreen test was easy (minus the more than 10 minutes it took to fill the vialthe instructions even offered suggestions on how to produce more spit like biting your tongue or cheeksinformation I never thought I'd need), but I admit, I was nervous to find out my results. Its easy to casually say I know exactly what I would do if it turns out Im a carrier for something horrible, but when it comes down to it, its a hard reality to face. But I was glad I chose to work with JScreen since they gave me my results on a call with a genetic counselor rather than in an email Id have to read and analyze on my own. During my results call I had a bunch of questions for my assigned counselor, Melanie Hardy, MS, MS, LCGC, the assistant director of genetic counseling services at JScreen (I am a journalist after all!). She told me that many couples who both test positive for carrying a condition find that the condition in question is mild and/or treatable.

Julia Wilkinson, the reproductive health genetic counselor at Invitae, also shared that its uncommon for both partners to carry variants in the same gene. More than 95 percent of couples tested in our lab find that even if one partner has a potentially concerning mutation, the other doesnt, so their overall risk of having a child with a genetic disorder is low.

Thats good to hear, but it led to my next big question: But what happens if I do carry something serious? For me, the next step would be to have my husband take a test as well, then we would have to consider our options

Hardy then told me that even if a couple learns they are carriers of a more severe condition, there are options for having a healthy child. The news may be surprising at first, but once they find that they have the support of the JScreen genetic counselors who will answer their questions and provide information and resources, they are then equipped with what they need to have a healthy family, she says. She explained the five options that couples have when they find out they are at an increased risk to having a child with a genetic condition:

1) In vitro fertilization with preimplantation genetic testing (PGT) of the embryo. To break it down simply, Hardy says that this process combines sperm and egg outside the womb, then the lab grows embryos and removes some cells from the embryos to complete genetic testing. Only those embryos that do not have the genetic condition get implanted.

2) Use of an egg or sperm donor (the egg or sperm donor will have been tested for the condition and found not to be a carrier).

3) Prenatal testing of the placenta (chorionic villus sampling or CVS) or amniotic fluid (amniocentesis) during pregnancy. This option comes with the caveat that the couple would have two decisions if the baby is found to be affected with the genetic condition: proceed with that information or end the pregnancy.

4) Adoption.

5) Test the child for the condition after birth and treat as needed or as available if the child has the genetic condition.

One common word kept coming up in my conversation with genetic counselors from JScreen and Invitae. Both called the testing process empowering." And I totally agree. We all do so many things before and during pregnancy to ensure the health of our future children: take prenatal vitamins, eat healthy food, stop drinking alcohol, go to routine doctor appointments; so why would we not do a checkup for our future babys genes too?

We cant determine if there is a reproductive risk without testing and avoiding testing doesnt prevent the possibility for devastating news about a child being affected, says Hardy. It is only with testing, preparation, and guidance that couples can make decisions to ensure a healthy family.

Continued here:
Why I Decided to Do Genetic Screening Before Trying to Get Pregnant - Yahoo Lifestyle

Recommendation and review posted by Bethany Smith

Preimplantation genetic testing or preimplantation genetic diagnosis is a technique in which the embryos prepared through in vitro fertilization are tested for defects before implantation. Preimplantation genetic testing enables physicians and to identify the defects present in the embryos and selectively implant healthy embryos in the uterus which increases the chances of delivering a genetically healthy baby. Preimplantation genetic testing helps people to avoid the hereditary disorders that prevail in the family to be carried into the baby. The preimplantation techniques involve various steps like the collection of eggs from the mother or egg donor which are later in vitro fertilized. Fertilized eggs are then tested for various genetic conditions through screening processes. Healthy embryos may be frozen and stored for further use whereas unfit embryos are destroyed. The healthy embryos are implanted to induce pregnancy. Preimplantation genetic testing also serves another purpose like gender selection. However, this application is currently facing several ethical questions. The preimplantation genetic testing is currently is gaining popularity as a fertility treatment option among carriers of sex-linked genetic disorders, single gene donors, people suffering from chromosomal disorders, older women seeking pregnancy and among women who experience recurring abortions.

Preimplantation Genetic Testing Market: Drivers & Restrains

Increasing awareness about preimplantation genetic testing among people suffering from genetic disorders is expected to drive demand for preimplantation genetic testing procedures According to the Global Genes Organization a non profit organization aimed towards promoting needs of the rare diseases community, there are nearly 7000 distinct rare diseases and genetic or rare diseases affect nearly 350 million people globally. Moreover according to the National Institutes of Health (NIH), about 50% people affected by rare diseases are children. Growing number of people suffering from genetic diseases are expected to increase demand for preimplantation genetic testing procedures in order to have a healthy child. Owing to high pregnancy chances with preimplantation genetic testing procedure as compared to other fertility treatments the demand for PGI testing is expected to witness high demand among people seeking IVF treatments. Increasing applications for preimplantation genetic testing for diagnosis of diseases like cancer and other minor disabilities like deafness is expected to create high growth opportunities for preimplantation genetic testing market stakeholders. Even though the preimplantation genetic testing market promises a health growth, restraints like the ethical issues related to preimplantation genetic testing and stringent regulatory policies may hamper the revenue growth of the market. Socio economic concerns related to sex determination and sex discrimination of the embryo are rising issues that might restraint the development of technology in preimplantation genetic testing market.

Preimplantation Genetic Testing Market: Segmentation

The global preimplantation genetic testing market is segmented into following key segments: by application type, by product type, by end users and by geography

Segmentation by application type

Aneuploidy Screening

Gender Screening

Chromosomal Aberration Screening

HLA Typing

Single Gene Disorder Screening

Others

Segmentation by product type

Instruments

Reagents

Analyzer Software

Accessories & Consumables

Segmentation by end user

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Hospitals

Diagnostic Laboratories

Academic Institutions

Fertility Clinics & Maternity Centers

Preimplantation Genetic Testing Market: Overview

The global preimplantation genetic testing market is expected to witness high growth over the forecast period owing to the increasing number of people seeking IVF treatment for fertility related problems and increasing awareness of preimplantation genetic testing being for avoiding birth defects among babies. Increasing adoption of preimplantation genetic testing in the developed and emerging countries is expected to create healthy growth opportunities for the market participants in the global preimplantation genetic testing market

Preimplantation Genetic Testing Market: Region wise Outlook

Geographically the global preimplantation genetic testing market is segmented into seven key regions: North America, Latin America, Western Europe, Eastern Europe, Asia Pacific excluding Japan (APEJ), Japan and Middle East and Africa (MEA).

Geographically North America and Western Europe are expected to dominate the market for preimplantation genetic testing owing to high awareness among the people and presence of several end users providing preimplantation genetic testing services. APEJ is expected to be the next lucrative market. Latin America and MEA regions are also expected to witness significant growth in the preimplantation genetic testing market. The growth of preimplantation genetic testing market is mainly dependent on resolving the ethical restraints involved and introduction of effective regulations for ethical use of technology in various regions.

Preimplantation Genetic Testing Market: Market Participants

Some players in the preimplantation genetic testing market are Abbott Laboratories, Natera, Inc., Illumina, Inc., Perkin Elmer, Inc. Thermo Fisher Scientific, Inc., F. Hoffmann-La Roche Ltd and others

The research report presents a comprehensive assessment of the market and contains thoughtful insights, facts, historical data, and statistically supported and industry-validated market data. It also contains projections using a suitable set of assumptions and methodologies. The research report provides analysis and information according to categories such as market segments, geographies, types, technology and applications.

The report covers exhaustive analysis on:

Market Segments

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Regional analysis includes:

North America (U.S., Canada)

Latin America (Mexico. Brazil)

Western Europe (Germany, Italy, France, U.K, Spain, Nordic countries, Belgium, Netherlands, Luxembourg)

Eastern Europe (Poland, Russia)

APEJ (China, India, ASEAN, Australia & New Zealand)

Japan

Middle East and Africa (GCC, S. Africa, N. Africa)

The report is a compilation of first-hand information, qualitative and quantitative assessment by industry analysts, inputs from industry experts and industry participants across the value chain. The report provides in-depth analysis of parent market trends, macro-economic indicators and governing factors along with market attractiveness as per segments. The report also maps the qualitative impact of various market factors on market segments and geographies.

Report Highlights:

Detailed overview of parent market

Changing market dynamics of the industry

In-depth market segmentation

Historical, current and projected market size in terms of volume and value

Recent industry trends and developments

Competitive landscape

Strategies of key players and product offerings

Potential and niche segments/regions exhibiting promising growth

A neutral perspective towards market performance

Must-have information for market players to sustain and enhance their market footprint

NOTE All statements of fact, opinion, or analysis expressed in reports are those of the respective analysts. They do not necessarily reflect formal positions or views of Future Market Insights.

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See more here:
Preimplantation Genetic Testing Market Growth in Technological Innovation, Competitive Landscape Mapping the Trends and Outlook - Wolf Mirror

Recommendation and review posted by Bethany Smith

Tickets to the Pulaski County Extension Homemakers annual tasting luncheon will go on sale Tuesday, October 1, at 8:00 o'clock at the Extension Office. This big event will be held on Friday, November 15, with seating at 12:00 noon. The cost is $20, which includes the tasting of lots of new foods and a recipe booklet of all items served. The seating to the luncheon is limited so be sure to buy your ticket or tickets soon. To make sure that all persons are treated equally, advanced sales or call in orders are not accepted. However one person can purchase as many tickets as needed starting Tuesday Morning, October 1. The "Pumpkin Kisses and Holiday Wishes" theme will be held at the Langdon Street Baptist Church Activity Center at 103 Langdon Street.

The Pulaski County Extension Homemakers Bazaar will be held at the Somerset Mall this year. The bazaar "Pumpkin Kisses and Harvest Wishes" will be held on Saturday, November 23, starting at 10:00 o'clock. Tables will be rented to those people wanting to sell their crafts, art work, pieced goods, baskets, quilts, wood crafts, knitting and crocheting items, etc. Tables are rented for $10 each to Homemaker members, or $25 each to non-members. Participants are not allowed to bring in their own table. Come by the Extension Office to rent your table.

Another homemaker year has begun. Extension Homemakers are encouraged to pay their dues to their secretary or treasurer who will turn in their dues to the Extension Office. Mail box members and other interested persons wanting to join the homemaker, can pay at the Pulaski County Extension Office. Dues are $11 yearly and membership is available to everyone.

If you are a female over 50 years of age do you have Ovarian Cancer Screening yearly? September is Ovarian Cancer Awareness Month. One of the reasons that ovarian cancer is so deadly is that in its early stages, it rarely causes any symptoms. Yet ovarian cancer causes more deaths each year than any other cancer of the female reproductive system. Ovarian cancer awareness and screening is of utmost importance for all females.

The Pulaski County Extension Homemakers, and all of the other 119 Kentucky County Extension Homemaker Groups, donate money every year to University of Kentucky Ovarian Screening program. This research program encourages all women over the age of 50 to have an Ovarian Screening yearly, and it is a free health screening. You don't have to be a member of the Extension Homemakers for this service. For information about UK Ovarian Cancer Screening Program, or to make an appointment, call 1-800-766-8279. Ovarian screening is also available at the Pulaski County Health Department, but you call the number listed above to make an appointment for Lexington or Somerset.

The American Cancer Society estimates that in 2019 about 23,000 women will get a new diagnosis of ovarian cancer and about 14,000 of them will die from it. In Kentucky the ASC estimates about 280 women will be newly diagnosed with ovarian cancer, and 190 will die from it. Women from every county in the state have participated in the screening. You may be one that needs to be participating too.

Ovarian cancer is deadly and sneaky. When detected early, it is often curable, but most women who have it don't have any symptoms until it has progressed to an advanced stage, when survival is unlikely. Screening and early detection are critical to saving lives.

The exact causes of ovarian cancer are unknown. We do know that the risk for developing is linked to several factors. Age is a major one with women 50 years of age and older being at a higher risk. Women who have a documented family history of ovarian or breast cancers are more likely to develop the disease. Through genetic testing, which you must request, if a woman has a BRCA1 or BRCA2 mutation she has a higher chance of developing both ovarian and breast cancers. (BRCA1: a gene that normally acts to restrain the growth of cells in the breast but which, when mutated, predisposes to breast cancer)

Other factors linked to the disease include an early age of beginning menstruation, late age at natural menopause, endometriosis, infertility or not bearing children, obesity, and hormone replacement therapy. A lowered risk of ovarian cancer also appears to be a benefit of both bearing children and breast-feeding.

How is ovarian cancer treated? Initial treatment is surgery to remove one or both ovaries and fallopian tubes, depending on the stage of the disease. Some patients may require a hysterectomy and some may need chemotherapy. The disease is highly curable if detected early so if you are over 50 years of age, begin your screening today. The University of Kentucky Ovarian Cancer Screening Program offers free screenings via transvaginal ultrasound to all Kentucky women over age 50 and women over 25 with a documented history of ovarian cancer.

In Kentucky there are six sites that offer this free screening from UK. Call 1-800-766-8279 to schedule your appointment. The Kentucky Extension Homemakers and the Telford Foundation provided the initial funding for the program, and continue to support it today.

Educational programs of Kentucky Cooperative Extension serve all people regardless of economic or social status and will not discriminate on the basis of race, color, ethnic origin, national origin, creed, religion, political belief, sex, sexual orientation, gender identity, gender expression, pregnancy, marital status, genetic information, age, veteran's status, or physical or mental disability.

It's fall and time for fall food. Enjoy this salad today.

Fall Harvest Salad

5 cups torn leaf lettuce

2 cups spinach leaves

1 medium red apple, chopped

1 medium pear, chopped

4 teaspoons lemon juice

cup dried cranberries

cup feta cheese crumbles

cup chopped walnuts

Dressing

2 tablespoons olive oil

2 tablespoons balsamic vinegar

1 teaspoons Dijon mustard

2 teaspoons honey

teaspoon salt

Will yield: 8 1 cup servings

Combine leaf lettuce and spinach leaves in a large salad bowl. Mix apples and pears with the lemon juice in a small bowl and add to the lettuce mixture. Prepare the dressing by whisking together the olive oil, balsamic vinegar, mustard, honey and salt. Pour over the lettuce mixture and toss to coat. Sprinkle the salad with cranberries, feta cheese and walnuts. Serve immediately.

Events at the Extension Office

Join Denise Salter at the Extension Office on Monday, September 23, at 10:00 o'clock for a free Card Making Class. Learn how to save money by making your own beautiful cards. This group will be meeting in the basement of the Extension Office, Room B.

The Pulaski County Extension Homemakers Council will meet on Monday, September 23, at 11:30 for their by-monthly meeting. Lunch will be provided.

Monday, September 23, you are invited to attend a class on "Addiction 101" at the Extension Office starting at 1:00 o'clock. Addiction to drugs or alcohol is one of the most complex, baffling and heartbreaking conditions in the world. Most people know at least one significant person in their lives who has been affected. Christy Guffey, the FCS Agent in Clinton County, will be conducting the class that is opened to all interested persons.

Just Among Friends Extension Homemakers Club will meet on Thursday, September 26, at 1:00 o'clock at the Extension Office.

Attention All Quilters: The First Baptist Church on Main Street in Somerset, Kentucky will be offering quilters the opportunity to have their antique quilts photographed and documented by the Kentucky Heritage Quilt Society. All documentation of antique quilts is stored at the Western Kentucky University. This service will be available Friday and Saturday, October 18 and 19 in the Bridge Area of the church. Call the Pulaski County Extension Office to schedule your appointment for this service at 679-6361.

Link:
'Holiday Tasting' will be Saturday, November 23 | Lifestyles - Commonwealth Journal's History

Recommendation and review posted by Bethany Smith

The Hinckley Institute Radio HourThis week on the program, we bring you a forum on the Abuelas de la Plaza de Mayo, a group formed in 1977 to locate and reunify with their grandchildren disappeared during the Argentinian dictatorship. This organization of women championed a matriarchal politics and began a legal, psychological and scientific movement to address the injustices and intergenerational traumas of the past. Critical to this effort was the combination of humanitarian justice, cutting edge genetic testing and international scientific exchanges that found 128 of the lost children.

This movement stands out as one in which the quest for human rights fueled scientific development and technological advancement. The genetic research conducted in Argentina would go on to advance the global study of genetic and forensic testing, popularized today by DNA testing companies like 23andMe and AncestryDNA. For their work in defense of human rights, the Abuelas de la Plaza de Mayo received the Flix Houphout-Boigny Peace Prize in Paris in September of 2011.

Giving the talk is Alexandra Minna Stern, Professor and Chair of American Culture, Professor in History, Womens Studies, Obstetrics and Gynecology in the College of Literature, Science and the Arts at the University of Michigan.

This forum was presented by the International Studies Programs Health, Medicine, and the Environment Lecture Series and made possible thanks to the support from the Center for Latin American Studies.

This forum was recorded on April 8, 2019.

Podcast: Play in new window | Download (57.0MB)

Subscribe: Apple Podcasts | Android |

Related

Read the rest here:
Genetics and Justice: DNA Identification Technologies in Post-Dictatorial Argentina - KCPW

Recommendation and review posted by Bethany Smith

Tracking nucleic acids in living cells

Fluorescence in situ hybridization (FISH) is a powerful molecular technique for detecting nucleic acids in cells. However, it requires cell fixation and denaturation. Wang et al. found that CRISPR-Cas9 protects guide RNAs from degradation in cells only when bound to target DNA. Taking advantage of this target-dependent stability switch, they developed a labeling technique, named CRISPR LiveFISH, to detect DNA and RNA using fluorophore-conjugated guide RNAs with Cas9 and Cas13, respectively. CRISPR LiveFISH improves the signal-to-noise ratio, is compatible with living cells, and allows tracking real-time dynamics of genome editing, chromosome translocation, and transcription.

Science, this issue p. 1301

We report a robust, versatile approach called CRISPR live-cell fluorescent in situ hybridization (LiveFISH) using fluorescent oligonucleotides for genome tracking in a broad range of cell types, including primary cells. An intrinsic stability switch of CRISPR guide RNAs enables LiveFISH to accurately detect chromosomal disorders such as Patau syndrome in prenatal amniotic fluid cells and track multiple loci in human T lymphocytes. In addition, LiveFISH tracks the real-time movement of DNA double-strand breaks induced by CRISPR-Cas9mediated editing and consequent chromosome translocations. Finally, by combining Cas9 and Cas13 systems, LiveFISH allows for simultaneous visualization of genomic DNA and RNA transcripts in living cells. The LiveFISH approach enables real-time live imaging of DNA and RNA during genome editing, transcription, and rearrangements in single cells.

Read the original:
CRISPR-mediated live imaging of genome editing and transcription - Science Magazine

Recommendation and review posted by Bethany Smith

The U.S. Patent and Trademark Office (USPTO) today awarded the University of California (UC), University of Vienna and Emmanuelle Charpentier a patent for CRISPR-Cas9 that, along with two others awarded this month, brings the teams comprehensive portfolio of gene-editing patents to 14.

Schematic representation of the CRISPR-Cas9 system. The Cas9 enzyme (orange) cuts the DNA (blue) in the location selected by the RNA (red). Image courtesy of Carlos Clarivan/Science Photo Library/NTB Scanpix

The newest patent, U.S. 10,415,061, covers compositions comprising single-molecule DNA-targeting RNAs or nucleic acids encoding single-molecule DNA-targeting RNAs, as well as methods of targeting and binding a target DNA, modifying a target DNA or modulating transcription from a target DNA with a complex that comprises a Cas9 protein and single-molecule DNA-targeting RNA.

On Sept. 10, the USPTO issued to the UC team U.S. patent 10,407,697 covering single-molecule guide RNAs or nucleic acid molecules encoding the guide RNAs. And on Sept. 3, the patent office issued U.S. patent 10,400,253, which covers compositions of single-molecule, DNA-targeting RNA (single-guide RNA, or sgRNA) and a Cas9 protein or nucleic acid encoding the Cas9 protein.

Another patent is set to issue next Tuesday, Sept. 24, bringing the total U.S. patent portfolio to 15. Three other patent applications have been allowed by the USPTO and are set to issue as patents in the coming months, which will raise the total to 18. These patents and applications span various compositions and methods for the CRISPR-Cas9 gene-editing technology, including targeting and editing genes and modulating transcription, and covering the technology in any setting, such as within plant, animal and human cells. The methods and compositions covered in UCs CRISPR-Cas9 portfolio come together to comprise the widest-ranging patent portfolio for the gene-editing technology.

This month, we have seen exponential growth of UCs U.S. CRISPR-Cas9 portfolio, said Eldora Ellison, Ph.D., lead patent strategist on CRISPR-Cas9 matters for UC and a director at Sterne, Kessler, Goldstein & Fox. We remain committed to expanding our robust portfolio to include additional methods and compositions for CRISPR-Cas9 gene editing so that the range of applications can be fully utilized for the benefit of humanity.

The team that invented the CRISPR-Cas9 DNA-targeting technology included Doudna and Martin Jinek at UC Berkeley; Charpentier, then at Umea University in Sweden and now director of the Max Planck Institute for Infection Biology in Germany; and Krzysztof Chylinski of the University of Vienna. The methods covered by todays patent, as well as the other methods claimed in UCs previously issued patents and those set to issue, were included among the CRISPR-Cas9 gene editing technology work disclosed first by the Doudna-Charpentier team in its May 25, 2012, priority patent application.

The 14 CRISPR-Cas9 patents in this teams portfolio are 10,000,772; 10,113,167; 10,227,611; 10,266,850; 10,301,651; 10,308,961; 10,337,029; 10,351,878; 10,358,658; 10,358,659; 10,385,360; 10,400,253; 10,407,697; and 10,415,061. These patents are not a part of the PTABs recently declared interference between 14 UC patent applications and multiple previously issued Broad Institute patents and one application, which jeopardizes essentially all of the Broads CRISPR patents involving eukaryotic cells.

International patent offices have also recognized the pioneering innovations of the Doudna-Charpentier team, in addition to the 14 patents granted in the U.S. so far. The European Patent Office (representing more than 30 countries), as well as patent offices in the United Kingdom, China, Japan, Australia, New Zealand, Mexico, and other countries, have issued patents for the use of CRISPR-Cas9 gene editing in all types of cells.

University of California has a long-standing commitment to develop and apply its patented technologies, including CRISPR-Cas9, for the betterment of humankind. Consistent with its open-licensing policies, UC allows nonprofit institutions, including academic institutions, to use the technology for non-commercial educational and research purposes.

In the case of CRISPR-Cas9, UC has also encouraged widespread commercialization of the technology through its exclusive license with Caribou Biosciences, Inc. of Berkeley, California. Caribou has sublicensed this patent family to numerous companies worldwide, including Intellia Therapeutics, Inc. for certain human therapeutic applications. Additionally, Dr. Charpentier has licensed the technology to CRISPR Therapeutics AG and ERS Genomics Limited.

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CRISPR portfolio now at 14 and counting - UC Berkeley

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Its been an alarming year for the worlds outlook on biodiversity. The Intergovernmental Science-Policy Platform on Biodiversity and Ecosystem Services (IPBES) put the world on notice that around 1 million species are facing extinction (PDF). A study published in August concluded that it would take New Zealand 50 million years to recover the diversity of bird species it has lost since human colonization. And, while headlines about an insect apocalypse may have been hyperbolic, insect biodiversity is decreasing, and its a problem.

As evidenced in the IPBES report, current conservation efforts have not been sufficient to stem biodiversity loss, so innovative solutions might be necessary to support the web of life that supports human existence. In 2012, scientists first described the gene editing capabilities of CRISPR, a molecular tool that can be used to make targeted, precise changes to the DNA of plants, animals and microbes.

Since then, scientists have proposed myriad ways to use the technology. But could it be a boon to biodiversity? Can it help researchers understand and preserve corals and their ecosystems? What about applications to diversify agriculture to shore up food security? Or to combat invasive species plaguing ecosystems around the world?

While many scientists are eager to discuss the possibilities of using CRISPR to preserve biodiversity, they are also cautious. The effects of human interventions are not always predictable, and once a gene-edited species is released into the wild, controlling any negative effects will be difficult. Toni Piaggio, a research scientist at the U.S. Department of Agriculture (USDA) National Wildlife Research Center, says researchers should "never entirely sip the Kool-Aid" when it comes to CRISPR. Instead, she says, they should "spend a lot of research time and intellectual energy" questioning themselves and their work.

While many scientists are eager to discuss the possibilities of using CRISPR to preserve biodiversity, they are also cautious.

Diversity for food security

But as millennia passed, domestication also decreased the genetic diversity within the plants we grow and eat. To understand why, imagine an ancient human 10,000 years ago, tired of smashing teosinte with rocks to get a few measly kernels out of their hard casings. If that person saw a plant with naked kernels exposed and available to eat without rock smashing they might select seeds from that plant to grow the next year. That works out great for the person, but the genetic diversity in the rest of the field is lost to future generations.

The same forces are at play today. When each tomato plant, for example, looks the same, grows at the same rate and produces pounds upon pounds of tomatoes, farming is easier and the food supply is more predictable if everything goes as usual.

Problem is, farming doesnt always follow usual, expected patterns. And climate change is increasing variability and unpredictability in agriculture. Many crops, as a result of their low genetic diversity, are not particularly well suited to cope with emerging climate patterns, leaving them susceptible to challenges such as drought, flooding or salty soils. So, says Lzaro Peres, a professor of plant physiology at the University of So Paulo, relying on a limited number of crop species to produce the worlds food is risky.

Peres and other researchers are trying to infuse agriculture with the genetic diversity of wild species. His research team started with a wild tomato and used CRISPR to edit a handful of key genes. Its goal was to make the versions of the genes in wild tomato look like the versions of the genes in domesticated tomato. In doing so, the wild tomato species gained some beneficial characteristics common to domesticated species. Through this process, de novo domestication, Peres and colleagues produced a tomato with more fruit, bigger fruit and more lycopene than wild tomatoes and that are genetically diverse from conventional domesticated tomatoes.

Does a change in plant size or color affect which insects are attracted to it? How does that affect the predators of those insects?

But, looking beyond a single crop into the ecosystem within which it exists is important, says Yolanda Chen, an associate professor in the College of Agriculture and Life Sciences at the University of Vermont. Chen studies the impact plant domestication can have on insect populations. She says that researchers need to consider how genes "operate within a broader community context" and not just in a single plant. Does a change in plant size or color affect which insects are attracted to it? How does that affect the predators of those insects?

Peres is mindful of the potential effects on agricultural ecosystems. Domesticating a wild tomato and growing it at scale could impact nuanced ecological relationships. Still, he says, he "sees mainly positive things" about the potential impacts of his work. "And one of the things is food security, because it is quite dangerous to depend on very few species for our food, feed and fiber."

Chen says that she thinks gene editing for de novo domestication is "less risky" than other genetic approaches, such as those that introduce entire new genes into a plant species. In de novo domestication, the edited versions of genes already exist in related domesticated tomato plants.

It likely will be a while before a new species of tomato developed to increase the genetic diversity of our food is available at the local grocery store. Peres says the work he and his group have published so far was a proof of concept; in other words, they showed that de novo domestication is feasible, but have no plan to commercialize that tomato. Theyve since turned their attention to a species of wild tomato from the Galpagos Islands that grows especially well in salty soils and is resistant to a white fly that can cause severe crop damage. If they are able to de novo domesticate this tomato, it could be used as an important crop for farmers dealing with salty, coastal soils.

In the end, Chen and Peres are both concerned about climate change, agriculture and biodiversity. They approach solutions to these concerns from different research perspectives, but both see diversity on the genetic and species levels in agricultural ecosystems as an important aspect of a food system that can withstand the challenges of climate change. In the future, domesticating new plant species potentially with gene editing might give farmers more options for growing diverse crops well-suited to specific climates.

Coral conservation

In 1770, British explorer Captain James Cook ran his ship, Endeavor, aground on the "insane labyrinth" that would become known as the Great Barrier Reef off the coast of Queensland, Australia. While Cook was credited with "discovering" the reef, coral reefs had been important to indigenous people for centuries before.

A few hundred years later, pollution and warming water have resulted in huge coral bleaching events around the world. While corals can survive bleaching, the stress does lead to increased mortality. Thats bad news for the marine species that inhabit corals. When corals are lost, reef ecosystems suffer, throwing the relationships between the thousands of species including fish, invertebrates, plants and turtles that live there out of balance.

Current conservation efforts for the worlds corals have been insufficient to curb bleaching events and sustain the valuable ecosystems corals support, according to the IPBES report. So there is a certain urgency to finding new approaches to conservation. A 2019 report by biologists laid out different conservation approaches and evaluated their potential risks and benefits. And with the 2018 announcement that scientists have used CRISPR to edit genes in coral, gene editing is seen as a potential strategy. Maybe.

Current conservation efforts for the worlds corals have been insufficient to curb bleaching events and sustain the valuable ecosystems corals support.

Marie Strader, now an assistant research professor at Auburn University, was a lead researcher as a graduate student on the international team of scientists that produced the work. The scientists edited three types of genes in a vibrantly colored coral called Acropora millepora. The goal of the editing was to "break" or mutate the genes, and in some larvae, it did.

As this proof-of-concept study was successful meaning they were able to edit the coral genes they targeted at least some of the time other researchers can use their methods as a blueprint for editing other genes in Acropora millepora and editing other coral species. For starters, Strader says, theyll likely look at genes involved in the coral life cycle and temperature sensitivity. Understanding those processes, Strader says, can "translate into conservation efforts down the line."

For example, researchers can use CRISPR in the lab to help them understand which genes are important for tolerance to warm waters. If they edit a gene in the lab and the resulting coral can better tolerate warm waters, according to Strader, the scientists could look at natural coral populations for those that naturally have that genetic mutation. Armed with that understanding, researchers might be more successful at conservation efforts such as breeding corals to help them keep their cool as the heat turns up.

If they edit a gene and the resulting coral can better tolerate warm waters, the scientists could look at natural coral populations for those that naturally have that genetic mutation.

For one thing, there are still plenty of technical obstacles. In Straders work, individual edited corals ended up with a mix of edited and unedited copies of the genes. To realize the full effect of a gene edit and to pass it down to future generations, each cell of the coral ideally should have the same edit. And other details, such as making sure CRISPR edits only the targeted gene or genes, "need to be worked out before it would be a viable option for conservation purposes," Strader says.

Furthermore, says John Bruno, a marine ecologist at the University of North Carolina at Chapel Hill, conservation efforts need to protect not just corals but also the thousands of other species that rely on them. According to Bruno, gene editing 10 or 20 species of corals to tolerate warm water just isnt enough. As "nobodys going to CRISPR all billion species that are in the ocean," he says, conservation needs to focus on the whole ecosystem and not just a few species. "The solution is rather obvious, just radically mitigate greenhouse gas emissions," he says acknowledging thats no easy feat.

Running interference

The situation with corals is "dire," according to Bruno. But even in coral species that have seen precipitous declines, often still many potentially on the order of millions of individuals are left, he says.

Back on shore, some animal populations are much smaller and easily could slip out of existence under the thumb of invasive species. In New Zealand, native birds evolved without mammalian predators. Many are large and flightless, so when mammals such as rats, possums and stoats arrived with humans, the birds were easy targets. According to one study, these invasive animals are responsible for the loss of an estimated 26.6 million chicks and eggs of native bird species each year.

Gene drives, which have become more plausible with the advent of gene editing, could offer a more humane way of managing invasive populations and protecting the species they endanger.

Gene drives, which have become more plausible with the advent of gene editing, could offer a more humane way of managing invasive populations and protecting the species they endanger.

"So many things have been done with the best possible intention, and we find that theres just been unforeseen consequences," says Helen Taylor, a conservation geneticist and honorary research fellow at the University of Otago. She points out that while possums are pests in New Zealand, they are an important species in Australia. If a possum with the New Zealand gene drive somehow were released in Australia, the effects could be devastating.

Maud Quinzin, a conservation geneticist and senior postdoctoral associate, recently began working in MITs Sculpting Evolution Lab with Kevin Esvelt, the scientist who first proposed CRISPR as a tool to create gene drives. Quinzin is using her understanding of ecosystem dynamics to help the Sculpting Evolution Lab think about the complex rippling effects of human interference in ecosystems.

Its important to look at the science from all angles, she says. "Developing gene-editing tools requires scientists with very different expertise sharing ideas and progress from early on in the process." For example, if an invasive rat species is eradicated from an island, will other species even other invasive species become more populous? "You have to think about the dynamic in that ecosystem," she says. Since suggesting that CRISPR could be used for gene drives, Esvelt himself has been vocal about his concerns.

Still, Quinzin has been on the front lines of conservation biology, watching populations of valued species go extinct, and shewants communities to be presented with all options for conservation. For scientists to present those options, though, they really need to understand the places where they might work, Quinzin says. That understanding comes not just from researchers, but also from the people who live in those places. "It is really important that you respect the values and the knowledge in a place," Quinzin says, including "not only the scientific information but also the indigenous or local knowledge." By engaging with local communities as technology develops, Quinzin says, researchers can focus on developing technology in ways that align with a communitys cultural, social, political and environmental values.

Moving forward

In the short term, agriculture might be the most likely use of CRISPR to protect biodiversity. In fact, the first gene-edited crop hit the market in the United States in early 2019. Individual countries are still figuring out how to regulate edited plants, with a big distinction being made between plants that could have emerged through natural mutations and plants containing larger edits, such as those containing new DNA.

At the very least, the work of scientists such as Peres could expand the genetic diversity of our crop plants, adding more options to the table as farmers, scientists and other stakeholders work toward a food-secure world. And having options is important. No single solution can save biodiversity everywhere. And carelessly applied solutions can cause more problems.

Scientists do seem to be proceeding with caution. At least some coral researchers decline to consider using CRISPR in the wild. Scientists studying gene drives are vocally pointing out the limitations of the technology and extolling the role nonscientists must play in the decisions to use or not use CRISPR for conservation purposes.

"I think we have a really big not just opportunity, but an obligation to get it out there in the public eye as much as possible," Piaggio says. And if scientists dont get public buy-in, they shouldnt use the technology, she says. "I think we have to be OK with that."

Quinzin says that she and other scientists in her group want guidance from the public. At the same time, she notes that CRISPR "could be such an amazing tool if we are respectful [and] responsible and use it properly."

There are no perfect or universal solutions to the biodiversity crisis the world is facing. And the causes cannot be forgotten in pursuit of an antidote. Thats why it will take scientists and conservationists with diverse approaches working in different areas to make a difference.

This article was originally published on Ensia.

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Can the gene editing technology CRISPR help reduce biodiversity loss worldwide? - GreenBiz

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A gene-editing technique that has shown promise as a potential cure for sickle-cell disease is now being tested in humans. But if it works, will the people who need it even be able to get it? Now that a cure may be in sight, this is an urgent question, says Vence Bonham, a senior advisor to the director of the National Human Genome Research Institute.

Sickle-cell disease (SCD) is a genetic blood disorder that affects millions of people in the world. It causes the production of abnormal red blood cells and can lead to intense pain, strokes, and organ and tissue damage.

From a scientific perspective, its an exciting time for people who suffer from the disease, Bonham said today at MIT Technology Reviews EmTech conference. Researchers are testing a technique that uses the precise gene-editing tool CRISPR to modify a single gene associated with the disease.

Justin Saglio

But from a sociological standpoint, argued Bonham, the work is just beginning. SCD is more common in certain ethnic groups, particularly people of African descent. And while there are around 100,000 people with the disease in the US, the vast majority live in sub-Saharan Africa and India, Bonham said.

He and colleagues recently conducted a study intended to explore the attitudes and beliefs toward the promising technique held among people with SCD, their family members, and their physicians. Many of the people Bonham and his colleagues spoke with expressed skepticism that a potential CRISPR-based cure would be affordable and accessible to those who need it. Although they did find renewed hope, they also observed cautionary, apprehensive undertones to this hope, which they concluded stem in part from decades of medical disenfranchisement of the SCD community.

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According to one physician interviewed for the study, there is a danger that other rare diseases that tend to affect people with more resources might get more attention and, potentially, funding. As a result, there is a concern that the SCD population could get left in the dust. This population is already skeptical, since they have been left in the dust with so many other things, the physician added.

Besides a cure itself, we also need better and cheaper ways to expand the benefits of this new technology, Bonham said. The potential is great, but we must ask the question: Who will benefit?

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CRISPR could help us cure sickle-cell disease. But patients are wary. - MIT Technology Review

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(more about Power Words)

cancerAny of more than 100 different diseases, each characterized by the rapid, uncontrolled growth of abnormal cells. The development and growth of cancers, also known as malignancies, can lead to tumors, pain and death.

Cas9An enzyme that geneticists are now using to help edit genes.It can cut through DNA, allowing it to fix broken genes, splice in new ones or disable certain genes. Cas9 is shepherded to the place it is supposed to make cuts by CRISPRs, a type of genetic guides. The Cas9 enzyme came from bacteria. When viruses invade a bacterium, this enzyme can chop up the germs DNA, making it harmless.

cellThe smallest structural and functional unit of an organism. Typically too small to see with the unaided eye, it consists of a watery fluid surrounded by a membrane or wall. asyeasts, molds, bacteria and some algae, are composed of only one cell.

clinicaltrialA research trial that involves people.

CRISPRAn abbreviation pronounced crisper for the term clustered regularly interspaced short palindromic repeats. These are pieces of RNA, an information-carrying molecule. They are copied from the genetic material of viruses that infect bacteria. When a bacterium encounters a virus that it was previously exposed to, it produces an RNA copy of the CRISPR that contains that virus genetic information. The RNA then guides an enzyme, called Cas9, to cut up the virus and make it harmless. Scientists are now building their own versions of CRISPR RNAs. These lab-made RNAs guide the enzyme to cut specific genes in other organisms. Scientists use them, like a genetic scissors, to edit or alter specific genes so that they can then study how the gene works, repair damage to broken genes, insert new genes or disable harmful ones.

disorder(in medicine) A condition where the body does not work appropriately, leading to what might be viewed as an illness. This term can sometimes be used interchangeably with disease.

DNA(short for deoxyribonucleic acid) Along, double-stranded and spiral-shaped molecule inside most living cells that carries genetic instructions. It is built on a backbone of phosphorus, oxygen, and carbon atoms. In all living things, from plants and animals to microbes, these instructions tell cells which molecules to make.

engineerA person who uses science to solve problems. As a verb, to engineer means to design a device, material or process that will solve some problem or unmet need.

gene(adj. genetic) A segment of DNA that codes, or holds instructions, for a cells production of a protein. Offspring inherit genes from their parents. Genes influence how an organism looks and behaves.

geneticHaving to do with chromosomes, DNA and the genes contained within DNA. The field of science dealing with these biological instructions is known as genetics. People who work in this field are geneticists.

hemoglobinA molecule that binds to oxygen in the blood, carrying it around to tissues.

immune(adj.) Having to do with the immunity. (v.) Able to ward off a particular infection.Alternatively, this term can be used to mean an organism shows no impacts from exposure to a particular poison or process. More generally, the term may signal that something cannot be hurt by a particular drug, disease or chemical.

insightThe ability to gain an accurate and deep understanding of a situation just by thinking about it, instead of working out a solution through experimentation.

multiplemyelomaThis cancer starts in a type of white blood cells known as plasma cells. Part of the immune system, they help guard the body from germs and other harmful substances.

muscleA type of tissue used to produce movement by contracting its cells, known as muscle fibers. Muscle is rich in protein, which is why predatory species seek prey containinglots of this tissue.

mutation(v. mutate) Some change that occurs to a gene in an organisms DNA. Some mutations occur naturally. Others can be triggered by outside factors, such as pollution, radiation, medicines or something in the diet. A gene with this change is referred to as a mutant.

nerveA long, delicate fiberthat transmits signalsacross the body of an animal. An animals backbone contains many nerves, some of which control the movement of its legs or fins, and some of which convey sensations such as hot, cold or pain.

neuronAn impulse-conducting cell. Such cells are found in the brain, spinal column and nervous system.

oxygenA gas that makes up about 21 percent of Earth's atmosphere. All animals and many microorganisms need oxygen to fuel their growth (and metabolism).

pharmaceuticalsMedicines, especially prescription drugs.

plasma (in medicine) The colorless fluid part of blood.

proteinA compoundmade from one or more long chains of amino acids. Proteins are an essential part of all living organisms. They form the basis of living cells, muscle and tissues; they also do the work inside of cells. Among the better-known, stand-alone proteins are thehemoglobin (in blood) and the antibodies (also in blood) that attempt to fight infections. Medicines frequently work by latching onto proteins.

redblood cellColored red by hemoglobin, these cells move oxygen from the lungs to all tissues of the body. Red blood cells are too small to be seen by the unaided eye.

retinaA layer at the back of the eyeball containing cells that are sensitive to light and that trigger nerve impulses that travel along the optic nerve to the brain, where a visual image is formed.

RNAA molecule that helps read the genetic information contained in DNA. A cells molecular machinery reads DNA to create RNA, and then reads RNA to create proteins.

sarcomaA family of more than 70 cancers that begin in bones or in connective tissues.

technologyThe application of scientific knowledge for practical purposes, especially in industry or the devices, processes and systems that result from those efforts.

therapy(adj. therapeutic) Treatment intended to relieve or heal a disorder.

variantA version of something that may come in different forms. (ingenetics) A gene having a slight mutation that may have left its host species somewhat better adapted for its environment.

wombAnother name for the uterus, the organ in mammals in which a fetus grows and matures in preparation for birth.

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Genetics CRISPR enters its first human trials - Science News for Students

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In a recent review, scientists highlight the potential of gene editing technologies like CRISPR/Cas9 to not only understand the molecular mechanisms behind Parkinsons disease, but also identify new targets for treatment.

The review study, Interrogating Parkinsons disease associated redox targets: Potential application of CRISPR editing, was published in the journal Free Radical Biology and Medicine.

One of the hallmarks of PD is the loss of dopamine-producing neurons in the substantia nigra a brain region involved in the control of voluntary movements, and one of the most affected in PD. This occurs due to the clustering of a protein called alpha-synuclein in structures commonly known as Lewy bodies inside neurons.

Parkinsons is complex and multifactorial disease, with both genetic and environmental factors playing a role in either triggering or exacerbating the disease.

Genetic causes can explain 10% of all cases of PD called familial PD , meaning that in the majority of the cases (sporadic PD) there is an interplay between genetics and environmental risk factors.

Researchers atSechenov Universityin Russia and theUniversity of Pittsburgh reviewed the role of metabolic pathways, especially problems with mitochondria cells powerhouses and iron accumulation, as well as mechanisms in cell death (called apoptosis and ferroptosis) in the development and progression of Parkinsons disease.

These processes were discussed in the context of genome editing technologies, namely CRISPR/Cas9 a technique that allows scientists to edit genomes, inserting or deleting DNA sequences, with precision, efficiency and flexibility.

CRISPR is a promising technology, a strategy to find new effective treatments to neurodegenerative diseases, Margarita Artyukhova, a student at the Institute for Regenerative Medicineat Sechenov and the study first author, said in a press release.

Mitochondria dont work as they should in people withPD, resulting in shortages of cellular energy that cause neurons to fail and ultimately die, particularlydopamine-producing neurons. Faulty mitochondria are also linked to the abnormal production of reactive oxygen species, leading to oxidative stressan imbalance between the production of free radicals and the ability of cells to detoxify them that also damages cells over time.

Because mitochondrial dysfunction is harmful, damaged mitochondria are usually eliminated (literally, consumed and expelled) in a process called mitophagy an important cleansing process in which two genes, called PINK1 and PRKN, play crucialroles. Harmful changes in mitophagy regulation is linked with neurodegeneration in Parkinsons.

Previous studies with animal models carrying mutations in the PINK1and PRKNgenes showed that these animals developed typical features of PD mitochondrial dysfunction, muscle degeneration, and a marked loss of dopamine-producing neurons.

PINK1codes for an enzyme that protects brain cells against oxidative stress, whilePRKNcodes for a protein called parkin. Both are essential for proper mitochondrial function and recycling by mitophagy. Mutations in both the PINK1 and PRKNgene have been linked with early-onset PD.

However, new research suggests that the role of PINK1 and PRKNin Parkinsons could be more complex and involve other genes likePARK7(DJ-1), SNCA (alpha-synuclein) andFBXO7 as well as a fat molecule called cardiolipin.

CRISPR/Cas9 genome editing technology may be used to help assess the role of different genetic players in Parkinsons disease, and to look for unknown genes associated with disease progression and development. Moreover, this technology can help generate animal and cellular models that might help scientists decipher the role of certain proteins in Parkinsons and discover potential new treatment targets.

Iron is another important metabolic cue in Parkinsons. While its essential for normal physiological functions, excessive levels of iron can be toxic and lead to the death of dopamine-producing neurons in the substantia nigra.

Iron may also interact with dopamine, promoting the production of toxic molecules that damage mitochondria and cause alpha-synuclein buildup within neurons.

CRISPR/Cas9 technology can be used to help dissect the role of proteins involved in iron transport inside neurons, which in turn may aid in designing therapies to restore iron levels to normal in the context of Parkinsons disease.

Finally, researchers summarized evidence related to the role of two cell death pathways ferroptosis and apoptosis in PD. Ferroptosis is an iron-dependent cell death mechanism by which iron changes fat (lipid) molecules, turning them toxic to neurons. This process has been implicated in cell death associated with degenerative diseases like Parkinsons, and drugs that work to inhibit ferroptosis have shown an ability to halt neurodegeneration in animal models of the disease.

Apoptosis refers to a programmed cell death mechanism, as opposed to cell death caused by injury. Both apoptosis and ferroptosis speed the death of dopaminergic neurons.

CRISPR/Cas9 may help to pinpoint the key players in cell death that promote the loss of dopaminergic neurons in Parkinsons disease, while understanding the array of proteins that are involved in these processes.

These insights into the mechanisms of PD pathology [disease mechanisms] may be used for the identification of new targets for therapeutic interventions and innovative approaches to genome editing, including CRISPR/Cas9, the researchers wrote.

Genome editing technology is currently being used in clinical trials to treat patients with late-stage cancers and inherited blood disorders, Artyukhova notes in the release.

These studies allow us to see vast potential of genome editing as a therapeutic strategy. Its hard not to be thrilled and excited when you understand that progress of genome editing technologies can completely change our understanding of treatment of Parkinsons disease and other neurodegenerative disorders, she adds.

Patricia holds a Ph.D. in Cell Biology from University Nova de Lisboa, and has served as an author on several research projects and fellowships, as well as major grant applications for European Agencies. She has also served as a PhD student research assistant at the Department of Microbiology & Immunology, Columbia University, New York.

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CRISPR/Cas9 Potential in Advancing Parkinson's Understanding and Treatment Focus of Review Study - Parkinson's News Today

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We are pleased to announce Technology Networks Explores the CRISPR Revolution. Through a series of exclusive interviews with world-renowned scientists and bioethicists, Technology Networks Explores the CRISPR Revolution will investigate the ground-breaking research taking place in the CRISPR space, CRISPR "controversies" and whether the CRISPR technology looks set to fulfil its promise of revolutionizing science.

The series will feature interviews with researchers behind the discovery of the CRISPR mechanism, such as Professor Francisco Mojica, the scientist involved its development as a gene-editing tool, Professor Jennifer Doudna, and the "godfather" of human genome research, Professor George Church.

The series will also explore the latest technologies available in the CRISPR "toolbox" including industry perspectives, its application in agriculture and farming through a conversation with Professor Alison Van Eenennaam and insights into the global conversation surrounding its ethical implications from Professor Glenn Cohen.

Kicking off the series on Oct 14th is an interview with the humble and immensely influential microbiologist, Professor Francisco Mojica.

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Technology Networks Explores the CRISPR Revolution Coming Soon - Technology Networks

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Duchenne muscular dystrophy (DMD) is a rare but devastating genetic disorder that causes muscle loss and physical impairments. Researchers at the University of Missouri School of Medicine have shown in a mouse study that the powerful gene editing technique known as CRISPR may provide the means for lifelong correction of the genetic mutation responsible for the disorder.

Children with DMD have a gene mutation that interrupts the production of a protein known as dystrophin. Without dystrophin, muscle cells become weaker and eventually die. Many children lose the ability to walk, and muscles essential for breathing and heart function ultimately stop working.

"Research has shown that CRISPR can be used to edit out the mutation that causes the early death of muscle cells in an animal model," said Dongsheng Duan, PhD, Margaret Proctor Mulligan Professor in Medical Research in the Department of Molecular Microbiology and Immunology at the MU School of Medicine and the senior author of the study. "However, there is a major concern of relapse because these gene-edited muscle cells wear out over time. If we can correct the mutation in muscle stem cells, then cells regenerated from the edited stem cells will no longer carry the mutation. A one-time treatment of the muscle stem cells with CRISPR could result in continuous dystrophin expression in regenerated muscle cells."

In collaboration with other MU colleagues and researchers from the National Center for Advancing Translational Sciences, Johns Hopkins School of Medicine and Duke University, Duan explored whether muscle stem cells from mice could be efficiently edited. The researchers first delivered the gene editing tools to normal mouse muscle through AAV9, a virus that was recently approved by the U.S. Food and Drug Administration to treat spinal muscular atrophy.

"We transplanted AAV9 treated muscle into an immune-deficient mouse," said Michael Nance, a MD-PhD program student in Duan's lab and the lead author of the paper. "The transplanted muscle died first then regenerated from its stem cells. If the stem cells were successfully edited, the regenerated muscle cells should also carry the edited gene."

The researchers' reasoning was correct, as they found abundant edited cells in the regenerated muscle. They then tested if muscle stem cells in a mouse model of DMD could be edited with CRISPR. Similar to what they found in normal muscle, the stem cells in the diseased muscle were also edited. Cells regenerated from these edited cells successfully produced dystrophin.

"This finding suggests that CRISPR gene editing may provide a method for lifelong correction of the genetic mutation in DMD and potentially other muscle diseases," Duan said. "Our research shows that CRISPR can be used to effectively edit the stem cells responsible for muscle regeneration. The ability to treat the stem cells that are responsible for maintaining muscle growth may pave the way for a one-time treatment that can provide a source of gene-edited cells throughout a patient's life."

With more study, the researchers hope this stem cell-targeted CRISPR approach may one day lead to long-lasting therapies for children with DMD.

Reference: Nance et al. 2019.AAV9 Edits Muscle Stem Cells in Normal and Dystrophic Adult Mice. Molecular Therapy.DOI: https://doi.org/10.1016/j.ymthe.2019.06.012.

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Researchers Use CRISPR to Correct Mutation in Duchenne Muscular Dystrophy Model - Technology Networks

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A team has used a lentiviral capsid-based bionanoparticle system to deliver CRISPR-Cas9 gene editing therapies, reducing undesired effects.

Researchers have developed an improved CRISPR delivery system for gene editing, through a lentiviral capsid system. The team say that their findings could be useful in research and clinical applications by improving safety and avoiding possible immune responses.

using a traditional lentiviral vector allows the bionanoparticle to efficiently and safely deliver CRISPR-Cas9

The team, from Wake Forest Institute of Regenerative Medicine (WFIRM), US, packaged the Cas9 protein and guide RNA together within a lentiviral capsid-based bionanoparticle system.

Previously, the two components had to be delivered separately which was not as convenient, said Dr Baisong Lu, assistant professor of regenerative medicine at WFIRM and one of the lead authors of the paper.

Conventional CRISPR-Cas9 is not completely accurate and could potentially cut unexpected locations, causing unwanted results.

However, the using a traditional lentiviral vector allows the bionanoparticle to efficiently and safely deliver CRISPR-Cas9. The researchers observed that it reduced off-target rates compared to regular CRISPR-Cas9.

A similar strategy should be translatable to other editor proteins for gene disruption, said Anthony Atala, MD, director of WFIRM and a co-author of the paper. We may be able to utilise this to package and deliver other RNPs into mammalian cells, which has been difficult to achieve so far.

The findings were published in Nucleic Acids Research.

See more here:
Researchers improve CRISPR-Cas9 delivery efficiency - Drug Target Review

Recommendation and review posted by Bethany Smith

UK-based Oxford Nanopore has obtained a licence to CRISPR-Cas9 IP for nanopore sequencing, a third-generation approach used in the sequencing of biopolymers.

Oxford Nanopore, which specialises in DNA/RNA sequencing technology, announced the non-exclusive licence agreement with biotech company Caribou Biosciences yesterday, September 19.

Caribou was founded by scientists from the University of California, Berkeley, including CRISPR pioneer Jennifer Doudna.

Gordon Sanghera, CEO of Oxford Nanopore, said: The Cas9 technique will enable users to select and isolate the regions of the genome they are most interested in, including those not available to existing methods, ready for rapid analysis using our long-read, real-time sequencing technology.

According to the company, Cas9 enrichment with Oxford Nanopore sequencing enables scientists to cost-effectively sequence targeted regions that were not accessible previously.

Sanghera added: The entire library preparation process takes less than two hours so if combined with our portable sequencer MinION, this has the potential to open up fast-turnaround, near-sample testing in new ways.

In October last year, Amgen invested 50 million ($66 million) in Oxford Nanopore, as part of Amgens focus on using human genetics to deliver new medicines to patients.

Earlier in 2018, Oxford Nanopore announced it had raised 100 million from global investors, to be used to support the companys next phase of commercial expansion, including a new high-tech manufacturing facility in Oxford.

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Oxford Nanopore, CRISPR-Cas9, Caribou Biosciences, Jennifer Doudna, gene-editing, genetics, nanopore, University of California,

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Oxford Nanopore signs CRISPR licence - Life Sciences Intellectual Property Review

Recommendation and review posted by Bethany Smith

At this time, there is no cure for Duchenne muscular dystrophy (DMD), although there is one treatment for a subgroup of the disease. That is Sarepta Therapeutics Exondys 51 for DMD patients with a confirmed mutation amenable to exon 51 skipping. Recently the U.S. Food and Drug Administration (FDA) rejected Sareptas golodirsen for DMD with a confirmed mutation appropriate for exon 53 skipping.

DMD is a muscle wasting disease caused by mutations in the dystrophin gene. It is a progressive disease that usually causes death in early adulthood, with serious complications that include heart or respiratory-related problems. It mostly affects boys, about 1 in every 3,500 or 5,000 male children.

There just might be, however, hope for an actual cure. Researchers at the University of Missouri-Columbia, utilized CRISPR gene editing in a mouse model, to edit out the gene mutation and transplant AAV9 treated muscle into the mice. The transplanted muscle cells carried the edited gene and successfully produced dystrophin, the protein that is not produced in sufficient quantities in DMD patients.

The dystrophin gene is the largest in the body, and codes for the dystrophin protein, which is involved in muscle development and activity. One of the reasons DMD has been a tough nut to crack is that because of the genes size, its too large to fit into the typical viral vectors used in gene therapies. Thats partially why Sareptas approach is to use a type of RNA splicing that forces cells to skip over the faulty section of genetic code. This results in a shortened (truncated) protein that is still functional.

Research has shown that CRISPR can be used to edit out the nutation that causes the early death of muscle cells in an animal model, said Dongsheng Duan, the Margaret Proctor Mulligan Professor in Medical Research in the Department of Molecular Microbiology and Immunology at the MU School of Medicine and senior author of the study.

However, Duan went on, there is a major concern of relapse because these gene-edited muscle cells wear out over time. If we can correct the mutation in muscle stem cells, then cells regenerated from the edited stem cells will no longer carry the mutation. A one-time treatment of the muscle stem cells with CRISPR could result in continuous dystrophin expression in regenerated muscle cells.

Duans research, in collaboration with others at MU as well as the National Center for Advancing Translational Sciences, Johns Hopkins School of Medicine and Duke University, looked at whether muscle stem cells in mice could be effectively edited. They used AAV9, an adeno-associated virus recently approved by the FDA to treat spinal muscular atrophy (SMA)Novartis Zolgensma, which is also the source of the controversy over the companys data manipulation scandal.

They started by delivering CRISPR to normal mouse muscle via AAV9.

We transplanted AAV9-treated muscle into an immune-deficient mouse, said Michael Nance, an MD-PhD program student in Duans lab and the lead author of the paper. The transplanted muscle died first then regenerated from its stem cells. If the stem cells were successfully edited, the regenerated muscle cells should also carry the edited gene.

That appeared to work. They then tested if the muscle stem cells in the mice of DMD could be edited with CRISPRthey were.

This finding suggests that CRISPR gene editing may provide a method for lifelong correction of the genetic mutation in DMD and potentially other muscle diseases, Duan said. Our research shows that CRISPR can be used to effectively edit the stem cells responsible for muscle regeneration. The ability to treat the stem cells that are responsible for maintaining muscle growth may pave the way for a one-time treatment that can provide a source of gene-edited cells throughout the patients life.

The research was published in the journal Molecular Therapy.

More:
CRISPR Research Might Lead to Cure for Duchenne Muscular Dystrophy - BioSpace

Recommendation and review posted by Bethany Smith

Just because a business does not make any money, does not mean that the stock will go down. For example, although software-as-a-service business Salesforce.com lost money for years while it grew recurring revenue, if you held shares since 2005, you'd have done very well indeed. Nonetheless, only a fool would ignore the risk that a loss making company burns through its cash too quickly.

Given this risk, we thought we'd take a look at whether CRISPR Therapeutics (NASDAQ:CRSP) shareholders should be worried about its cash burn. In this article, we define cash burn as its annual (negative) free cash flow, which is the amount of money a company spends each year to fund its growth. The first step is to compare its cash burn with its cash reserves, to give us its 'cash runway'.

Check out our latest analysis for CRISPR Therapeutics

You can calculate a company's cash runway by dividing the amount of cash it has by the rate at which it is spending that cash. In June 2019, CRISPR Therapeutics had US$428m in cash, and was debt-free. In the last year, its cash burn was US$133m. That means it had a cash runway of about 3.2 years as of June 2019. Importantly, though, analysts think that CRISPR Therapeutics will reach cashflow breakeven before then. If that happens, then the length of its cash runway, today, would become a moot point. Depicted below, you can see how its cash holdings have changed over time.

NasdaqGM:CRSP Historical Debt, September 21st 2019

CRISPR Therapeutics boosted investment sharply in the last year, with cash burn ramping by 61%. That's bad enough, but the operating revenue drop of 96% points to a period of uncertainty and, quite potentially, heightened risk for holders." In light of the above-mentioned, we're pretty wary of the trajectory the company seems to be on. While the past is always worth studying, it is the future that matters most of all. So you might want to take a peek at how much the company is expected to grow in the next few years.

Even though it seems like CRISPR Therapeutics is developing its business nicely, we still like to consider how easily it could raise more money to accelerate growth. Companies can raise capital through either debt or equity. Commonly, a business will sell new shares in itself to raise cash to drive growth. We can compare a company's cash burn to its market capitalisation to get a sense for how many new shares a company would have to issue to fund one year's operations.

CRISPR Therapeutics has a market capitalisation of US$2.6b and burnt through US$133m last year, which is 5.1% of the company's market value. That's a low proportion, so we figure the company would be able to raise more cash to fund growth, with a little dilution, or even to simply borrow some money.

It may already be apparent to you that we're relatively comfortable with the way CRISPR Therapeutics is burning through its cash. For example, we think its cash runway suggests that the company is on a good path. Although we do find its falling revenue to be a bit of a negative, once we consider the other metrics mentioned in this article together, the overall picture is one we are comfortable with. It's clearly very positive to see that analysts are forecasting the company will break even fairly soon After considering a range of factors in this article, we're pretty relaxed about its cash burn, since the company seems to be in a good position to continue to fund its growth. For us, it's always important to consider risks around cash burn rates. But investors should look at a whole range of factors when researching a new stock. For example, it could be interesting to see how much the CRISPR Therapeutics CEO receives in total remuneration.

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Of course CRISPR Therapeutics may not be the best stock to buy. So you may wish to see this freecollection of companies boasting high return on equity, or this list of stocks that insiders are buying.

We aim to bring you long-term focused research analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material.

If you spot an error that warrants correction, please contact the editor at editorial-team@simplywallst.com. This article by Simply Wall St is general in nature. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. Simply Wall St has no position in the stocks mentioned. Thank you for reading.

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CRISPR Therapeutics (NASDAQ:CRSP) Is In A Strong Position To Grow Its Business - Yahoo Finance

Recommendation and review posted by Bethany Smith

CRISPR is the buzzword of the moment in the drug discovery industry mainly due to its potential to correct disease-causing mutations. However, those using the technology need to be mindful that it is used responsibly, and possible risks are considered before use. Mark Behlke discusses the potential of CRISPR in R&D and the challenges that it presents for researchers.

CRISPR TECHNOLOGY has generated much excitement in the drug discovery realm for its ability to make precise, permanent changes to DNA in animals, as first demonstrated approximately six years ago. It is currently being evaluated in early phase clinical trials for several disorders. Diseases caused by a single gene mutation sickle cell disease (SCD), Huntingtons disease and cystic fibrosis are all prime targets for using CRISPR gene therapy to correct the disease-causing DNA mutations. CRISPR is also being investigated as a treatment for acquired immune deficiency syndrome (AIDS) and to improve anti-tumour immunotherapy.

While news about potential CRISPR therapeutics and the start of new clinical trials dominate headlines in the lay press, CRISPR has also become a leading research tool to help scientists better understand gene function and establish model systems of human diseases needed to translate basic scientific knowledge into new medical treatments.

Link:
The elegant parallel of using CRISPR to understand disease mechanisms - Drug Target Review

Recommendation and review posted by Bethany Smith

-New data demonstrate successful differentiation of CRISPR-edited human pluripotent stem cells to pancreatic precursor cells-

ZUG, Switzerland, CAMBRIDGE, Mass., and SAN DIEGO, Sept. 17, 2019 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (CRSP), and ViaCyte, Inc., a privately-held cell therapy company, today presented data from the Companies regenerative medicine program targeted towards type 1 diabetes (T1D) in an oral presentation at the 55th Annual Meeting of the European Association for the Study of Diabetes (EASD) in Barcelona, Spain. The data demonstrate that the CyT49 pluripotent stem cell line, which has been shown to be amenable to efficient scaling and differentiation, can be successfully edited with CRISPR. The CyT49 pluripotent stem cell line is currently being used to generate islet progenitors for clinical trials.

These data provide further evidence that the combination of regenerative medicine and gene editing has the potential to offer durable, curative therapies to patients in many different diseases, including common chronic disorders like insulin-requiring diabetes, said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. We look forward to advancing our T1D program in partnership with ViaCyte.

We are pleased with the data presented at EASD, which bring us potentially one step closer to a transformational therapy for patients with insulin-requiring diabetes through the development of an immune-evasive gene-edited version of our technology, said Paul Laikind, Ph.D., Chief Executive Officer and President of ViaCyte. ViaCyte has led the field over the past decade, being the first group to demonstrate a number of essential milestones on the path to a broadly applicable cell replacement therapy for diabetes. Now, in partnership with CRISPR Therapeutics, we aim to achieve yet another first, the development of an immune-evasive cell replacement therapy as a potential cure for T1D. The work being presented at EASD is an important step along that path.

To protect pancreatic islet cells from immune rejection, researchers utilized CRISPR/Cas9 gene editing to generate CyT49 clones that lack the 2-microglobulin (B2M) gene, a required component of the major histocompatibility complex class I (MHC-I), and express a transgene encoding programmed death-ligand 1 (PD-L1) to further protect from T-cell attack. Edited clonal cells maintained karyotypic stability and showed in vitro protection against T-cell mediated cell lysis.

About the CRISPR-ViaCyte CollaborationCRISPR Therapeutics and ViaCyte entered into a strategic collaboration in 2018 focused on the discovery, development, and commercialization of novel regenerative medicines including gene-edited allogeneic stem cell-derived therapies for the treatment of diabetes. The Companies are currently evaluating a preclinical-stage therapeutic candidate for insulin-requiring diabetes including type 1 diabetes, for which the Companies will jointly assume responsibility for development and commercialization worldwide.

About CRISPR TherapeuticsCRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer AG, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in London, United Kingdom. For more information, please visit http://www.crisprtx.com.

About ViaCyteViaCyte is a privately-held regenerative medicine company developing novel cell replacement therapies as potential long-term diabetes treatments to achieve glucose control targets and reduce the risk of hypoglycemia and diabetes-related complications. ViaCytes product candidates are based on the derivation of pancreatic isletprogenitor cells from pluripotent stem cells, which are then implanted in durable and retrievable cell delivery devices. Over a decade ago, ViaCyte scientists were the first to report on the production of pancreatic cells from a stem cell starting point and the first to demonstrate in an animal model of diabetes that, once implanted and matured, these cells secrete insulin and other pancreatic hormones in response to blood glucose levels. ViaCyte has two product candidates in clinical-stage development. The PEC-Direct product candidate delivers the pancreatic isletprogenitor cells in a non-immunoprotective device and is being developed for type 1 diabetes patients who have hypoglycemia unawareness, extreme glycemic lability, and/or recurrent severe hypoglycemic episodes. The PEC-Encap (also known as VC-01) product candidate delivers the same pancreatic isletprogenitor cells in an immunoprotective device and is being developed for all patients with diabetes, type 1 and type 2, who use insulin. ViaCyte is also developing immune-evasive stem cell lines, from its proprietary CyT49 cell line, which have the potential to further broaden the availability of cell therapy for diabetes and other potential indications. ViaCyte is headquartered in San Diego, California. ViaCyte is funded in part by the California Institute for Regenerative Medicine (CIRM) and JDRF. For more information, please visit http://www.viacyte.com.

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CRISPR Forward-Looking StatementThis press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding CRISPR Therapeutics expectations about any or all of the following: (i) the safety, efficacy and clinical progress of our various clinical programs including CTX001 and CTX110; (ii) the status of clinical trials (including, without limitation, the timing of filing of clinical trial applications and INDs, any approvals thereof and the timing of commencement of clinical trials), development timelines and discussions with regulatory authorities related to product candidates under development by CRISPR Therapeutics and its collaborators; (iii) the number of patients that will be evaluated, the anticipated date by which enrollment will be completed and the data that will be generated by ongoing and planned clinical trials, and the ability to use that data for the design and initiation of further clinical trials; (iv) the intellectual property coverage and positions of CRISPR Therapeutics, its licensors and third parties as well as the status and potential outcome of proceedings involving any such intellectual property; (v) the sufficiency of CRISPR Therapeutics cash resources; and (vi) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words believes, anticipates, plans, expects and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: the potential for initial and preliminary data from any clinical trial (including CTX001 and CTX110) not to be indicative of final trial results; the risk that the initial data from a limited number of patients (as is the case with CTX001 at this time) may not be indicative of results from the full planned study population; the outcomes for each CRISPR Therapeutics planned clinical trials and studies may not be favorable; that one or more of CRISPR Therapeutics internal or external product candidate programs will not proceed as planned for technical, scientific or commercial reasons; that future competitive or other market factors may adversely affect the commercial potential for CRISPR Therapeutics product candidates; uncertainties inherent in the initiation and completion of preclinical studies for CRISPR Therapeutics product candidates; availability and timing of results from preclinical studies; whether results from a preclinical trial will be predictive of future results of the future trials; uncertainties about regulatory approvals to conduct trials or to market products; uncertainties regarding the intellectual property protection for CRISPR Therapeutics technology and intellectual property belonging to third parties, and the outcome of proceedings (such as an interference, an opposition or a similar proceeding) involving all or any portion of such intellectual property; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics most recent annual report on Form 10-K, and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at http://www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

CRISPR Investor Contact:Susan Kim+1 617-307-7503susan.kim@crisprtx.com

CRISPR Media Contact:Jennifer PaganelliWCG on behalf of CRISPR+1 347-658-8290jpaganelli@wcgworld.com

ViaCyte Investor Contact: Matthew LaneGilmartin Group on behalf of ViaCyte, Inc. +1 617-901-7698matt@gilmartinir.com

ViaCyte Media Contact:Jessica Yingling, Ph.D. Little Dog Communications Inc. on behalf of ViaCyte, Inc. +1 858-344-8091jessica@litldog.com

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CRISPR Therapeutics and ViaCyte Present Positive In Vitro Data Towards a Potential Immune-Evasive Cell Replacement Therapy for Diabetes at EASD 2019 -...

Recommendation and review posted by Bethany Smith

CRISPR may be a cure, but clinical trials may lack volunteers because of black patients' mistrust of biased and unethical medical practices.

"It's like something stabbing you in your bone just like repeatedly. And you can't stop it. And... it's something that makes you really tense, you just can't move... It's been a part of my life since I was six months old."

Twenty-three-year-old Maiya Washington has been living with sickle cell, a life-long "invisible" disease. It's a disorder with no visibly detectable signs and not often talked about.

"Although it is a disease that is invisible, so to speak, we go through a lot, you know, and we go through a lot to be normal to live life to get jobs to go to school. And it's hard. It's really hard," says Washington.

Sickle Cell Disease impacts about100,000 Americans,mostly African Americans. It's a genetic defect that affects red blood cells turning normal round red blood cells into sickle shapes. That shape can cause clumping andblock oxygen and blood flow,which can then lead to a wide range of health issues likestrokes, kidney problems,and organ failure.

"It's really debilitating, honestly," says Washington. "And it's hard because... you need somebody there with you. It's not like you can take yourself to the hospital. Or if you're at home and you're trying to manage the pain by yourself, you still need somebody there with you, because you're not able to do the simple things like go to the restroom by yourself, get yourself some water, fix yourself something to eat"

Currently, the only option to cure sickle cell disease is a bone marrow transplant, also called a stem cell transplant. But it requires a matching donor, which can be difficult to find. But now, the gene-editing tool CRISPR may bypass the need for a match and serve as a cure for most people. Dr. John Tisdale has worked on another Sickle Cell Disease gene therapy at the National Institute of Health.

"And it's all coming from red blood cells with a single misspelling, in their hemoglobin. So we should be able to fix that it's just one base," says Tisdale.

Related StoryIs The Machine That Can Snip And Swap Our DNA Awesome Or Ominous?

Here's how it works. First, stem cells are pulled from the patient's body. CRISPR is used to edit the DNA, and the new, edited stem cells are then re-inserted back into the body. The hope is that it will generate healthy red blood cells. Changes to the DNA won't be passed down to future children.

Could this development lead to a cure for sickle cell? A clinical trial using CRISPR can help determine that. So far, testing on 12 people has already begun. Biopharmaceutical company Vertex has partnered with CRISPR Therapeutics and they're looking for morevolunteers for this clinical trial.

Dr. Alexis Thompson, Head of Hematology, Lurie Childrens Hospital of Chicago says, "I certainly am very excited about it as a provider, is that patients will have choices. There is not a one size fits all for sickle cell disease, or really, really for in a community For the first time, there will be multiple, multiple clinical trials that are opening and hopefully those will lead to new treatments... because much of what we learn from sickle cell will be rapidly applicable to other conditions."

But as exciting as the potential for a cure for Sickle Cell Disease is, finding enough volunteers for testing is really difficult. Sure, CRISPR poses risks, including death. But one of the biggest barriers isblack patients' mistrust of the medical community,so much so that clinical trialstend to lack black patient enrollment.This is based on racist treatment both in the past and present.Science writer Usha Lee MacFarlingpoints to unethical medical practices of the past, like the use of Henrietta Lacks' cells without permission and leaving syphilis untreated in hundreds of black men in the Tuskegee experiment.

"I think there's just a huge awareness in the black community of these studies that were, you know, racist, that really treated black women of color and poor women as guinea pigs. It's a very sharp pain And it's definitely affecting people's reluctance, and inability to trust, the largely white medical establishment," says MacFarling.

And today, some black patients say that bias persists in medicine. Because sickle cell patientsvisit the emergency room an average of three times a year, they're often assumed to be addicts for seeking drugs to ease their pain.

Related StoryResearchers' Gene Technology Removes HIV From Mice For The First Time

"Doctors [have]...given me an inappropriate amount of medicine. That wasn't helping, that kind of basically looked at me as like, you know, drug-seeking, or just like faking it," says Washington.

In spite of all this, Maiya says she wants to participate in the trial because of how excruciating her pain is.

"I feel as though most people who deal with sickle cell or any kind of disability that alters their quality of life, they're going to be willing to figure out anything to get rid of what they had," says Washington.

In order to build trust between black sickle cell patients and CRISPR researchers, organizations like the American Society of Hematology and the Minority Coalition for Precision Medicine are doing community outreach. That includes even teaming up with churches.

Michael Friend, co-founder of Minority Coalition for Precision Medicine says: "It was kind of very easy to talk to faith-based leaders about sickle cell disease because it's a disease that primarily affects African Americans. And it's a disease that we found prevalent in most churches, and most pastors were familiar with the disease."

Related StoryScientists Concerned Over Program That Enlists Bugs To Spread Viruses

For now, every month Maiya's gets a blood transfusion to ease the pain. Her baby is lucky -- she doesn't have sickle cell, because her father doesn't have the gene.

"I wake up every day and she's there. She's my best friend and I love seeing her watching her grow so far, says Washington.

Most sickle cell patients don't live past their 40's and Maiya does worry about her future.

She says, "it does concern me because I want to be here as long as possible for her. And hoping that, you know, there's something that can come up that can be permanent, you know, as in terms of a cure or medication...just to help us have a longer lifespan and live a better quality of life. Because I do want to be able to see her grow up."

Continue reading here:
CRISPR Cure For Sickle Cell May Be Slowed By Black Patients' Mistrust - Newsy

Recommendation and review posted by Bethany Smith

We are at war with the mosquito. A swarming and consuming army of 110tn enemy mosquitoes patrols every inch of the globe except for Antarctica, Iceland and a handful of French Polynesian micro-islands. The biting female warrior of this droning insect population is armed with at least 15 lethal and debilitating biological weapons, to be used against 7.7 billion humans deploying suspect and often self-detrimental defensive capabilities. In fact, our defence budget for personal shields, sprays and other means of deterring her unrelenting raids is $11bn (8.8bn) a year, and rising rapidly. And yet her deadly offensive campaigns and crimes against humanity continue with reckless abandon. While our counterattacks are reducing the number of casualties she perpetrates malaria deaths in particular are declining rapidly the mosquito remains the deadliest hunter of human beings on the planet.

Taking a broad range of estimates into account, since 2000, the average annual number of human deaths caused by the mosquito was around 2 million. Humans came in a distant second at 475,000, followed by snakes (50,000), dogs and sandflies (25,000 each), the tsetse fly, and the assassin or kissing bug (10,000 each). The fierce killers of lore and Hollywood celebrity were much further down our list. The crocodile was ranked 10th, with 1,000 annual deaths. Next on the list were hippos with 500, and elephants and lions with 100 fatalities each. The much-slandered shark and wolf shared 15th position, killing an average of 10 people per annum.

Yet the mosquito does not directly harm anyone. It is the toxic and highly evolved diseases she transmits that cause an endless barrage of desolation and death. Without her, however, these sinister pathogens could not be transferred or vectored to humans, nor could they continue their cyclical contagion. In fact, without her, these diseases would not exist at all.

Our immune systems are finely tuned to our local environments. Mosquitoes do not respect international borders. Marching armies, inquisitive explorers and land-hungry colonists brought new diseases to distant lands, but were also brought to their knees by micro-organisms in the foreign lands they intended to conquer. As the mosquito transformed the landscapes of civilisation, humans were unwittingly required to respond to her universal projection of power. After all, the truth is that, more than any other external participant, the mosquito, as our deadliest predator, drove the events of human history to create our present reality.

It has been one of the most universally recognisable and aggravating sounds on Earth for 190m years the whine of a mosquito. After a long day of walking while camping with your family or friends, you quickly shower, settle into your lawn chair, open an ice-cold beer and exhale a deep, contented sigh. Before you can enjoy your first satisfying swig, however, you hear that all-too-familiar sound, signalling the approach of your soon-to-be tormentors.

It is nearing dusk, her favourite time to feed. Although you heard her droning arrival, she gently lands on your ankle without detection, as she usually bites close to the ground. It is always a female, by the way. She conducts a tender, probing, 10-second reconnaissance, looking for a prime blood vessel. With her backside in the air, she steadies her crosshairs and zeros in with six sophisticated needles. She inserts two serrated mandible cutting blades (much like an electric carving knife, with two blades shifting back and forth), and saws into your skin, while two other retractors open a passage for the proboscis, a hypodermic syringe that emerges from its protective sheath. With this straw she starts to suck out 3-5 mg of your blood, immediately excreting its water while condensing its 20% protein content. All the while, a sixth needle is pumping in saliva that contains an anticoagulant, preventing your blood from clotting at the puncture site. This shortens her feeding time, lessening the likelihood that you feel her penetration and splat her across your ankle. The anticoagulant causes an allergic reaction, leaving an itchy bump as her parting gift. The mosquito bite is an intricate and innovative feeding ritual required for reproduction. She needs your blood to grow and mature her eggs.

Please dont feel singled out. She bites everyone. There is absolutely no truth to the persistent myths that mosquitoes fancy females over males, that they prefer blonds and redheads over those with darker hair, or that the darker or more leathery your skin, the safer you are from her bite. It is true, however, that she does play favourites and feasts on some more than others. Blood type O seems to be the vintage of choice over types A and B, or their blend. People with blood type O get bitten twice as often as those with type A, with type B falling somewhere in between. (Disney/Pixar must have done their homework when portraying a tipsy mosquito ordering a Bloody Mary, O-positive in the 1998 movie A Bugs Life.) Those who have higher natural levels of certain chemicals in their skin, particularly lactic acid, also seem to be more attractive. From these elements, she can analyse which blood type you are. These are the same chemicals that determine an individuals level of skin bacteria and unique body odour. While you may offend others and perhaps yourself, in this case, being pungently rancid is a good thing, for it increases bacterial levels on the skin, which makes you less alluring to mosquitoes except for stinky feet, which emit a bacterium that is a mosquito aphrodisiac. The mosquito is also enticed by deodorants, perfumes, soap and other applied fragrances.

She also has an affinity for beer drinkers. Wearing bright colours is also not a wise choice, since she hunts by both sight and smell the latter depending chiefly on the amount of carbon dioxide exhaled by the potential target. So all your thrashing and huffing and puffing only magnetises mosquitoes and puts you at greater risk. She can smell carbon dioxide from 200 feet away. When you exercise, you emit more carbon dioxide through frequency of breath and output. You also sweat, releasing those appetising chemicals, primarily lactic acid, that invite the mosquitos attention. Lastly, your body temperature rises an easily identifiable heat signature. On average, pregnant women suffer twice as many bites, as they respire 20% more carbon dioxide, and have a marginally elevated body temperature. This is bad news for the mother and the foetus when it comes to infection from Zika and malaria.

Unlike their female counterparts, male mosquitoes do not bite. Their world revolves around two things: nectar and sex. Like other flying insects, when they are ready to mate, male mosquitoes assemble over a prominent feature in the landscape from chimneys to antennas to trees to people. Many of us grumble and flail in frustration as that dogged cloud of bugs droning over our heads shadows us when we walk, refusing to disperse. Take it as a compliment. Male mosquitoes have graced you with the honour of being a swarm marker. Mosquito swarms have been photographed extending 1,000 feet into the air, resembling a tornado funnel cloud. With the cocksure males stubbornly assembled over your head, females will fly into their horde to find a suitable mate. While males will mate frequently in a lifetime, one dose of sperm is all the female needs to produce numerous batches of offspring. She stores the sperm and dispenses them piecemeal for each separate birthing of eggs. Her short moment of passion has provided one of the two necessary components for procreation. The only ingredient missing is your blood.

Back at the campsite, you have just finished your strenuous hike, and proceed to the shower, where you lather up with soap and shampoo. After drying off, you apply body spray and deodorant before finally putting on your bright red-and-blue beachwear.

It is nearing dusk dinnertime for the Anopheles mosquito. You have done everything in your power to lure a famished female of the species. Having just mated in a swarming frenzy of eager male suitors, she willingly takes the bait and makes off with a few drops of your blood a blood meal three times her own body weight. She quickly finds the nearest vertical surface and, with the aid of gravity, continues to evacuate the water from your blood. Using this concentrated blood, she will develop her eggs over the next few days. She then deposits roughly 200 floating eggs on the surface of a small pool of water that has collected on a crushed beer can that was overlooked during cleanup as you and your party headed home. She always lays her eggs in water, although she does not need much. From a pond or stream to a minuscule puddle in the bottom of an old container, used tire or backyard toy, any will suffice.

Our mosquito will continue to bite and lay eggs during her one-to-three-week lifespan. While she can fly up to two miles, she rarely ranges more than 400 metres from her birthplace. Although it takes a few days longer in cool weather, given the high temperatures, her eggs hatch into wiggling, water-bound worms within two or three days. Skimming the water for food, they quickly turn into upside-down, comma-shaped tumbling caterpillars who breathe through two trumpets protruding from their water-exposed buttocks. A few days later, a protective encasement splits and healthy adult mosquitoes take flight, with a new generation of succubus females ready to feed. This maturation to adulthood takes roughly one week.

Bacteria, viruses and parasites, along with worms and fungi, have triggered untold misery, and have commanded the course of human history. Why have these pathogens evolved to exterminate their hosts? If we can set aside our bias for a moment, we can see that these microbes have journeyed through the natural selection voyage just as we have. This is why they still make us sick and are so difficult to eradicate. You may be puzzled: it seems self-defeating and detrimental to kill your host. The disease kills us, yes, but the symptoms of the disease are ways in which the microbe conscripts us to help it spread and reproduce. It is dazzlingly clever, when you stop to think about it. Generally, germs guarantee their contagion and replication prior to killing their hosts. Some, like the salmonella food poisoning bacteria and various worms, wait to be ingested that is, one animal eating another animal.

There is a wide range of waterborne transmitters, including giardia, cholera, typhoid, dysentery and hepatitis. Others, including the common cold, the 24-hour flu and true influenza, are passed on through coughing and sneezing. Some, such as smallpox, are transferred directly or indirectly by lesions, open sores, contaminated objects or coughing. My personal favourites strictly from an evolutionary standpoint, of course are those that covertly ensure their reproduction while we intimately ensure our own. These include the full gamut of microbes that trigger sexually transmitted diseases. Many sinister pathogens are passed from mother to foetus in utero.

Others that germinate typhus, bubonic plague, Chagas and trypanosomiasis (African sleeping sickness) catch a free ride provided by a vector (an organism that transmits disease) such as fleas, mites, flies, ticks and mosquitoes. To maximise their chances of survival, many germs use a combination of more than one method. The diverse collection of symptoms, or modes of transference, assembled by micro-organisms helps them effectively procreate and ensures the existence of their species. These germs fight for their survival just as much as we do, and stay an evolutionary step ahead of us as they continue to morph and shape-shift to circumvent our best means of extermination.

To understand the stealthy, sprawling influence of the mosquito on history and humanity, it is first necessary to appreciate the animal itself, and the diseases it transmits. According to a quotation erroneously attributed to Charles Darwin: It is not the strongest of the species that survives, nor the most intelligent that survives. It is the one that is most adaptable to change. Regardless of the origin of this passage, the mosquito and its diseases most notably malaria parasites are the quintessential example of the point it is making. They are masters of evolutionary adaptation. Mosquitoes can evolve and adapt to their changing environments within a few generations. During the Blitz of 1940-41, for example, as German bombs rained down on London, isolated populations of Culex mosquitoes were confined to the air-raid tunnel shelters of the London Underground, along with the citys resilient citizens. These trapped mosquitoes quickly adapted to feed on mice, rats and humans instead of birds, and are now a species distinct from their above-ground parental ancestors.

What should have taken thousands of years of evolution was accomplished in less than 100. In another 100 years, jokes Richard Jones, former president of the British Entomological and Natural History Society, there may be separate Circle line, Metropolitan line and Jubilee line mosquito species in the tunnels below London.

While the mosquito is miraculously adaptable, it is also a purely narcissistic creature. Unlike other insects, it does not pollinate plants in any meaningful way, or aerate the soil, or ingest waste. Contrary to popular belief, the mosquito does not even serve as an indispensable food source for any other animal. She has no purpose other than to propagate her species, and perhaps to kill humans. As the apex predator throughout our odyssey, it appears that her role in our relationship is to act as a countermeasure against uncontrolled human population growth.

Throughout our existence, the mosquitos toxic twins of malaria and yellow fever have been the prevailing agents of death and historical change, playing the role of antagonists in the protracted chronological war between man and mosquito.

Following that fateful mosquito bite, the miscreant malaria parasite will mutate and reproduce inside your liver for one to two weeks, during which time you will show no symptoms. A toxic army of this new mutated form will then explode out of your liver and invade your bloodstream. The parasites attach to your red blood cells, penetrate the outer defences, and feast on the haemoglobin within. Inside the cell, they undergo another metamorphosis and reproductive cycle. Engorged blood cells eventually burst, spewing both a duplicate form, which marches forward to attack fresh red blood cells and also a new asexual form that relaxedly floats in your bloodstream, waiting for mosquito transportation.

The parasite is a shape-shifter, and it is precisely this genetic flexibility that makes it so difficult to eradicate or suppress with drugs or vaccines. You are now gravely ill with an orderly, clockwork progression of chills followed by a mercury-driving fever that may touch 41C. This full-blown cyclical malarial episode has you in its firm grip, and you are at the mercy of the parasite. Lying prostrate and agonisingly helpless on sweat-soaked sheets, you twitch and fumble, curse and moan. You look down and notice that your spleen and liver are visibly enlarged, your skin has the yellowing patina of jaundice and you vomit sporadically. Your fever will relapse at precise intervals with each new burst and invasion of the parasite from your blood cells. The fever then subsides while the parasite eats and reproduces inside new blood cells.

The parasite uses sophisticated signalling to synchronise its sequencing, and this entire cycle adheres to a very strict schedule. The new asexual form transmits a chemical bite me signal in our blood, significantly boosting the chances of being picked up by a mosquito from an infected human to complete the reproductive cycle. Inside the stomach of the mosquito, these cells mutate once more, into both male and female varieties. They quickly mate, producing threadlike offspring that make their way out of the gut and into the salivary glands of the mosquito. Within the saliva glands, the malaria parasite shrewdly manipulates the mosquito to bite more frequently by suppressing the production of her anticoagulant and thus minimising her blood intake during a single feeding. This forces her to bite more frequently to get her required fill. In doing so, the malaria parasite ensures that it maximises its rate and range of transfer, its procreation and its survival.

Temperature is an important element for both mosquito reproduction and the life cycle of malaria. Given their symbiotic relationship, they are also both climate-sensitive. In colder temperatures, it takes longer for mosquito eggs to mature and hatch. Mosquitoes are also cold-blooded and, unlike mammals, cannot regulate their own body temperatures. They simply cannot survive in environments below 10C. Mosquitoes are generally at their prime health and peak performance in temperatures above 23C. A direct heat of 40C degrees will boil mosquitoes to death. For temperate, non-tropical zones, this means that mosquitoes are seasonal creatures with breeding, hatching and biting taking place from spring through autumn. Although never seeing the outside world, malaria needs to contend with both the short lifespan of the mosquito and temperature conditions to ensure replication. The timeframe of malaria reproduction is dependent on the temperature of the cold-blooded mosquito, which itself is dependent on the temperature outside. The colder the mosquito, the more sluggish malaria reproduction becomes, eventually hitting a threshold. Between 15C and 21C (depending on the type of malaria), the reproductive cycle of the parasite can take up to a month, exceeding the average life span of the mosquito. By then, she is long dead, and brings malaria down with her.

Warmer climates can sustain year-round mosquito populations, promoting endemic circulation of her diseases. Abnormally high temperatures can cause seasonal epidemics of mosquito-borne diseases in regions where they are generally absent or fleeting. Global warming also allows the mosquito and her diseases to broaden their topographical range. As temperatures rise, disease-carrying species, usually confined to southern regions and lower altitudes, creep north and into higher elevations.

Since a breakthrough discovery by a team led by the biochemist Dr Jennifer Doudna at the University of California, Berkeley in 2012, the revolutionary gene-editing innovation known as Crispr has shocked the world and altered our preconceived notions about our planet and our place on it.

The pages of many widely read magazines and journals are currently consumed by the topic of Crispr and mosquitoes. First successfully used in 2013, Crispr is a procedure that snips out a section of DNA sequencing from a gene and replaces it with another desired one, permanently altering a genome, quickly, cheaply, and accurately.

The Bill and Melinda Gates Foundation has been funding research into mosquito-borne diseases since its creation in 2000. In 2016 it made investments in Crispr mosquito research totalling $75m. Our investments in mosquito control, said the foundation, include nontraditional biological and genetic approaches as well as new chemical interventions aimed at depleting or incapacitating disease-transmitting mosquito populations. These genetic approaches include the use of Crispr machinery to eradicate mosquito-borne diseases, most notably malaria.

The strategic goal of the Gates Foundation is the extermination of malaria and other mosquito-borne diseases; it is not to bring the mosquito which is harmless when flying solo, untethered from a hitchhiking micro-organism to the brink of extinction. Of the more than 3,500 mosquito species, only a few hundred are capable of vectoring disease. Prefabricated, genetically modified mosquitoes rendered incapable of harbouring the parasite (a hereditary trait passed down their bloodline) might just end the timeless scourge of malaria. But, as Doudna and the Gates Foundation are aware, gene-swapping technology also has the potential to unleash darker, more sinister genetic blueprints with dangerous possibilities. Crispr research is a global phenomenon, and neither Doudna nor the foundation has a monopoly on its limitless designs, its instruments of implementation or its operational execution.

It has been dubbed the extinction drive, as this is precisely what it can accomplish the extermination of mosquitoes by way of genetic sterilisation. This theory has been floating around the scientific community since the 1960s. Crispr can now put these principles into practice. To be fair, the mosquito altered our DNA in the form of sickle cell and other genetic malarial safeguards; perhaps it is time to return the favour. Male mosquitoes that have been genetically modified with domineering selfish genes using Crispr are released into mosquito zones to breed with females to produce stillborn, infertile or only male offspring. The mosquito would be extinct in one or two generations. With this war-winning weapon, humanity would never again have to fear the bite of a mosquito. We would awaken to a brave new world, one without mosquito-borne disease.

An alternative is simply to make mosquitos harmless, a strategy supported and funded by the Gates Foundation. With gene drive technology, Gates explained in October 2018, essentially, scientists could introduce a gene into a mosquito population that would either suppress the population or prevent it from spreading malaria. For decades, it was difficult to test this idea. But with the discovery of Crispr, the research became a lot easier. And just last month, a team from the research consortium Target Malaria announced that they had completed studies where mosquito populations were fully suppressed. To be clear: the test was only in a series of laboratory cages filled with 600 mosquitoes each. But it is a promising start.

Dr Anthony James, a molecular geneticist at the University of California, Irvine, Crisprd a species of Anopheles mosquito to make it incapable of spreading malaria, by eliminating the parasites as they are processed through the mosquitos salivary gland. We added a small package of genes, explains James, that allows the mosquitoes to function as they always have, except for one slight change they can no longer harbour the malaria parasite.

The Aedes breed is more difficult to tackle, since it transmits a handful of diseases that include yellow fever, Zika, West Nile, chikungunya, Mayaro, dengue and other encephalitides. What you need to do is engineer a gene drive that makes the insects sterile, James said of the Aedes breed. It doesnt make sense to build a mosquito resistant to Zika if it could still transmit dengue and other diseases.

We have valid, although yet unknown, reasons to be careful what we wish for. If we eradicate disease-vectoring mosquito species, would other mosquito species or insects simply fill the ecological niche? What effect would eliminating mosquitoes have on natures biological equilibrium? What would happen if we exterminate species that play an essential but unrecognised role in our ecosystem? We are just beginning to ask these morally fraught and biologically ambiguous questions, and for now, no one really knows the answers.

This is an edited extract from The Mosquito: A Human History of Our Deadliest Predator by Timothy Winegard, published by Text on 26 September and available at guardianbookshop.co.uk

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Original post:
People v mosquitos: what to do about our biggest killer - The Guardian

Recommendation and review posted by Bethany Smith

The gene drive is one of the latest of advancements in genetic modification of living things. It may also be the most controversial, in a field that has seen more than its fair share of controversy. Traditionally, coverage of genetic modifications in food products has resembled war correspondence:

Side 1Gene drives are a valuable tool for controlling pests and perpetuating beneficial genes through agricultural productsSide 2Gene drives are a dangerous, untested and unnatural genetic changes created to deliberately drive a species to extinction.

Gene drive is a version of gene editinga newer, more precise way to change a DNA (or RNA) sequence, in this case by combining a guide RNA with an enzyme that can make a splice in the exact place where a sequence can be removed, another sequence inserted, or the existing sequence altered. Gene drive takes this to another level, making sure that a new or altered genetic sequence has a greater than 50 percent chance of being inherited. This can be done in a number of ways, some of which already exist in nature, some which are no different than traditional gene editing using CRISPR-Cas9, and others that have triggered a backlash from environmental activist groupsnon-governmental organizations (NGOs) that utilize fear for their own political ends.

So far, this debate has pitted NGOs like Friends of the Earth, Greenpeace and ETC Group (who are opposed to any genetic manipulations in food crops and animals) against scientists, some agricultural companies and even some government regulators (almost all of whom conclude that these products are no more dangerous than those developed through traditional breeding).

The activist effort is part of a long-standing campaign to conflate gene drives, gene editing and traditional transgenics (GMO) as the same technology, with the same scientific certainty (or uncertainty) and risks. In 2016, FOE and others asked for a worldwide moratorium on gene drives:

Gene drives, developed through new gene-editing techniques, are designed to force a particular genetically engineered trait to spread through an entire wild population potentially changing entire species or even causing deliberate extinctions. The statement urges governments to put in place an urgent, global moratorium on the development and release of the new technology, which poses serious and potentially irreversible threats to biodiversity, as well as national sovereignty, peace and food security.

Many times, these activist organizations have claimed the public shares their concerns. Citing survey data, the science community has retorted that most people embrace biotechnology when they recognize that it benefits them directly. But what has been missing from the battle between the pro- and anti-GMO positions is a scientific measure of public opinion on more recent techniques such as gene drive. In 2016, a comprehensive National Academies of Science (NAS) report called for not only continued research on the effectiveness and usefulness of gene drives, but also their ecological risks and engagement with the public. While institutions like FOE and ETC Group objected to the existence of gene drives, they did not represent the opinion of the public.

For the first time, that opinion was actually tested, by researchers at North Carolina State University and the University of Wisconsin. In a paper published in Science Advances, Zack Brown, assistant professor of resource economics at NC State and his colleagues surveyed 1,000 American adults on their opinions of gene drives. What they found, instead of opposition, was support for the technology, with a few caveats:

The survey results could be valuable in this early stage of gene drive (or gene editing, for that matter) development as research could possibly be directed toward designing drive strategies that could incorporate controlsnot an easy thing to do, Brown said in a press release.

This is the right timewhile the technology is still under development and before any release decisions have been madeto gain insights into what the public thinks, what types of information they prioritize from researchers, and who is trusted to carry out this sensitive research, said Michael Jones, a graduate student at NC State and co-author of the published survey results.

Another significant finding in the NC State/Wisconsin study was that Americans surveyed trusted universities and the US Department of Agriculture (USDA) (60 percent) over foreign universities, the US Department of Defense (18 percent) and private companies (16 percent) to research gene drive systems.

The survey did not ask respondents for their trust levels of NGOs like FOE and Greenpeace.

However, in another recently published survey, this one in Current Research in Biotechnology, 113 experts (scientists, government officials, agribusiness professionals) found that gene-edited crops posed little to no risk to society, the economy, human health or the environment. Less than five percent thought the techniques posed a high risk.

The experts, most of whom observed that NGOs generally opposed gene editing, gene drive and any other genetic modification, noted that this opposition is based on speculative risks, those that have no established theory or evidence data. The authors of the study, based at the University of Saskatchewan in Canada, warned that the problem with attempting to reconcile speculative risks with risks grounded in theory and evidence, is that speculative risks can be very fluid and dynamic, changing at will and [frequently] at the whim of eNGO political motives.

These new studies seem to support the idea that consumers are less wary of biotechnology when they know how its being deployed. While public opinion surveys show support with some caveats about taking precautions against accidents and outbreaks, by and large members of the public trust scientists, particularly those in academia and at relevant regulatory agencies to navigate this controversial but promising field of research.

Andrew Porterfield is a writer and editor, and has worked with numerous academic institutions, companies and non-profits in the life sciences.BIO. Follow him on Twitter@AMPorterfield

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Viewpoint: Public supports CRISPR, gene drives to battle infectious disease, plant pestsdespite activist opposition - Genetic Literacy Project

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Scientists are reporting the first use of the gene-editing tool CRISPR to try to cure a patients HIV infection by providing blood cells that were altered to resist the AIDS virus.

The gene-editing tool has long been used in research labs, and a Chinese scientist was scorned last year when he revealed he used it on embryos that led to the birth of twin girls. Editing embryos is considered too risky, partly because the DNA changes can pass to future generations.

Wednesdays report in the New England Journal of Medicine, by different Chinese researchers, is the first published account of using CRISPR to treat a disease in an adult, where the DNA changes are confined to that person.

The attempt was successful in some ways but fell short of being an HIV cure.

Still, it shows that gene editing holds promise and seems precise and safe in this patient so far, said Dr. Carl June, a University of Pennsylvania genetics expert who wrote a commentary in the journal.

Thats really good for the field, June said.

Chinese government grants paid for the research, which was done openly with advance notice on a scientific registry and standard informed consent procedures. Some of those steps were missing or questioned in last years embryo work.

There are no ethical concerns on this one, June said.

Gene editing permanently alters DNA, the code of life. CRISPR is a relatively new tool scientists can use to cut DNA at a specific spot.

The new case involves a 27-year-old man with HIV who needed a blood stem cell transplant to treat cancer. Previously, two other men were apparently cured of both diseases by transplants from donors with natural resistance to HIV because they have a gene mutation that prevents HIV from entering cells.

Since donors like this are very rare, the Chinese scientists tried to create similar HIV resistance by editing that gene in blood cells in the lab to try to mimic the mutation.

The transplant put the mans cancer in remission, and the cells that were altered to resist HIV are still working 19 months later. But they comprise only 5 percent to 8 percent of such blood cells, so theyre outnumbered by ones that can still be infected.

They need to approach 90 percent or more, I think, to actually have a chance of curing HIV, June said.

Scientists are testing various ways to make the gene editing more efficient, and our results show the proof of principle for this approach, one study leader, Hongkui Deng of Peking University in Beijing, wrote in an email.

One very encouraging result: multiple tests show that the editing did not have unintended effects on other genes.

One of the concerns is that they could make a Frankenstein cell, that they would hit other genes instead of the intended target, so its good that this did not happen, June said.

China appears to be moving fast on such research and may get treatments approved sooner than the United States, June said. He has financial ties to some gene therapy companies and is leading a different study testing CRISPR to fight cancer in the U.S. Three patients have been treated so far and some results are expected by the end of this year.

Several other U.S. studies have been trying to control HIV by altering patients own blood cells using a different gene-editing tool called zinc finger nucleases. The first such test began a decade ago in the U.S.

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Gene-editing tool shows promise in fight against HIV - The Columbian

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