Initially a Niche Market for Very Few with Small Population Impact

The first genetic tests directly available to consumers for health were offered in 1996. The concept, then, was both audacious and bold: the idea that individuals could explore their own human genome without the aid of a health care provider to order the test or interpret the results. Some consumer

The world has changed. In the last two years, personal genomics usage has exploded

advocates praised the development as empowering, while many medical and public health experts advised caution, given the lack of evidence that results were clinically useful and that the risk for potential harms was unknown. Meanwhile, the direct-to-consumer (DTC) genetic health test industry grew relatively slowly during the first two decades of its existence. During that time, personal genomics for any purpose was often perceived as a mere curiosity purchased by only a few wealthy individuals.

The world has changed. As reported in the May 22, 2018 Science News special report, personal genomics usage has exploded from what might have once been considered an unlikely source: mushrooming consumer interest in genealogy. Genealogy has grown dramatically to become the second most popular hobby in the United States and the second-most popular internet surfing topic. In recent years, costs for genetic ancestry tests have dropped dramatically and demand has responded in kind. Generally, the tests focus on identifying genetic variants important to ones cultural and geographic heritage and are not thought to involve health issues though it is conceivable they could raise important questions. For example, if one discovered previously unknown Ashkenazi Jewish ancestry, additional risk for certain genetic conditions might be discussed with ones physician.

Recent sales for DTC genetic health tests increased dramatically when several DTC test providers began bundling their popular DNA genealogy package with their health package. We find DTC tests for health concerningconducted as they often are without the involvement of a healthcare provider and without an understanding of clinical validity and utilityas we have detailed in our blogs: Think Before You Spit, Think (Again) Before You Spit, and Think After You Spit. Despite these valid concerns, bundled ancestry and health packages have been selling like hotcakes. While specific sales data for health related DTC tests are not publicly available, we can easily guess their significance. In late 2017, a bundled genealogy and health DNA test was one of Amazons top five Black Friday sellers. Meanwhile, overall, genetic tests for ancestry have continued to skyrocket. The total number of people who have taken direct to consumer genealogy tests was reported to have increased two fold in 2017 with the total number of people who have participated at greater than 12 million and rising.

Many questions come to mind that require better population level data to answer.

As the number of people who have participated in DTC genetic tests rises into the millions, these questions are becoming increasingly important to answer as a public health priority. What data we do have about consumer knowledge on genetic tests provides further reason for concern. A recent study based on an online survey of 1,001 adults representative of the population, found that public awareness of genomics and personalized medicine was not increasing in line with advancements in the industry. Seventy-three percent of the survey respondents had not heard of genetic counseling which is conducted by certified health professionals to advise consumers/patients on how to interpret genetic test results.

We need to know a lot more. We could better understand these issues by including questions about DTC genetic test awareness, usage, and impact in population-based surveysan approach used successfully in the past. (Examples include those reported by Jacobellis in 2004, Goddard in 2009, Kolor in 2012, and Agurs-Collins in 2015.)

With current and expanded data on use and impact of DTC genetic health tests, we can take steps to empower consumers to make more informed choices about their health behaviors and health resource expenditures. These measures could include providing:

One thing has become clear: DTC genetic tests, including those for health purposes, are now mainstream. Both before and after deciding to purchase, it is essential that the general public understands the potential harms and benefits of applications marketed or interpreted for health relevance. An important role for public health is to provide unbiased evidenced-based information. The CDC Office of Public Health Genomics will continue to provide regularly updated and searchable data on DTC tests in our Public Health Genomics Knowledge Base (PHGKB). Additional information on this topic can be found on our website and our Genomics and Health Weekly Update. In future blog posts, we will further explore the implications of consumer genetic testing on the health of individuals and populations.

As always, we welcome your comments and questions.

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Consumer Genetic Testing Is Booming: But What are the ...

Recommendation and review posted by Bethany Smith

Genetic Testing

If you or your partner have experience with genetic disorders in your family or simply need the security that comes from utilizing the best resources available, it is important to consider the use of genetic testing during your treatment. Not only does this state-of-the-art technology make IVF safer, as we are reducing the risk of pregnancy loss, but it also reduces the chance (and cost) of multiple cycles since we may ensure transfer of only the healthiest embryos. Genetic tests are performed on embryos to ensure the health of the chromosomes. Normally, there are 24 chromosomes (22 autosomes and an X and a Y chromosome).

The availability of genetic testing also allows our center to highlight the benefits of our less is more philosophy, which focuses on single embryo transfers to reduce the chance of multiple pregnancies. Transferring multiple embryos and resulting multiple pregnancies/births are stressful on the uterine environment, significantly decreasing the chance of a healthy pregnancy and increasing the chance of premature births.

It is common for patients to ask about the difference between PGD and PGS. The difference is significant and yet subtle. The purpose of PGD is to diagnose abnormal embryos to ensure that they are not transferred back into your uterus and improve your chances of having a healthy baby. PGD can only be run if you know that you or your partner are carriers of a genetic disorder. A special probe will be created to test for the specific disorder(s) that a couple is known to have. PGS on the other hand will screen for and identify unknown chromosomal abnormalities. This is better for patients who have a history of miscarriages or failed IVF cycles due to unknown circumstances. Most of our patients undergo PGS or Preimplantation Genetic Screening, but be sure to ask which one is right for you.

Genetic testing is very safe for both you and your embryos. We are not making designer babies, so there are no ethical issues to worry about. Some couples are concerned that the procedure will affect their chances of pregnancy, or more importantly, the health of their embryo. Because the procedure is done so early in the developmental process, at a time when cells from the embryo can potentially be removed, our genetic testing does not cause any harm to the developing embryo. Genetic testing is a vital resource for many couples, especially those with known family histories of genetic defects.

PGD/PGS/NGS can offer genetic screeningfor numerous diseases and disorders classified as either chromosomal disorders, single gene defects, or sex-linked disorders. Specific chromosomes are tested for specific disorders, including (but not limited to):

*PLEASE ASK OUR STAFF WHICH TESTS ARE RECOMMENDED FOR YOU*

New Hope Fertility embryologists can also test for X-linked diseases, which only affect males, (e.g. Hemophilia A, Adrenoleukodystrophy, Hunters disease) by identifying the sex of embryos and transferring only female embryos. We also offer PGD for single gene defects such as Cystic Fibrosis (CF), the common deletion (^F508), Spinal Muscular Atrophy (SMA), and Myotonic Dystrophy (DM).

NHFC also performs aneuploidy screening and chromosome translocations to detect abnormalities that may cause spontaneous abortions in early pregnancy. We can perform PGD for all single gene defects where the specific mutation is identified and as long as we can develop a special genetic probe for the disease.

New Hope Fertility Center of New York City is among the top NYC fertility clinics brings together a team of world-class, best fertility specialists that are committed to bring you the best of tomorrows IVF treatment, today. Our NYC fertility center named the Top Clinic of 2017 and is on top of the Forbes list of fertility centers in US. Dr. Zhang has been named among New Yorks Top Doctors

(Click the links below for more infertility information)

More here:
Preimplantation Genetic Diagnosis, PGS Testing, PGD Testing

Recommendation and review posted by Bethany Smith

Dear Reader,

This article is about information that most DNA test companies arent expecting you to readDid you know that the market for DNA tests has become fiercely competitive in the last year?In fact, the market has more than doubled in size. More people took a DNA test in 2017 than in ALL previous years combined! 2018 is expected to be another record-shattering year.

Its no surprise that DNA tests are in high demand. The things you learn from them are irresistible:

Ancestry Everything weve ever known about our heritage has come from our parents and grandparents. A DNA test can tell you so much more about who you are and where youre from. Most companies offer this basic service, but some are far better than others.

Family History This kind of goes hand-in-hand with Ancestry. If youre trying to sort out family mysteries, a DNA test can help you solve them. If your parents also have their DNA tested, you can sometimes sort out which genes you received from each side of the family.

Community Finding out your genetic heritage gets you a deeper connection to the places that youre from. On top of this, some of the best DNA test companies will connect you with people who share pieces of DNA with you. Many people use this feature to discover long-lost relatives.

Health Risks Some DNA tests can reveal unique traits embedded in your genetic code that may put you at risk for certain health conditions. This can help you alter your lifestyle to try and prevent them.

Family Planning A DNA test can help you find out what genes you may pass onto your children, for better or worse.

With so many people clamoring to get their hands on the benefits listed above, more and more DNA test companies have been rising to meet them. Unfortunately, theyre not all created equal.

Now its harder than ever to find a good DNA test

Luckily, youre in the right place. Our team of scientists, researchers, and writers at Genetics Digest know the field better than most. Weve examined nearly every DNA test in the booming market. Well help you sort out the great ones from the cheap knockoffs.

With that said, lets get started on Common Mistakes People Make When Shopping for a DNA Test

Mistake #1: Dont buy a brand by how popular it appears to be.

Some brands have a great marketing team with a massive advertising budget. You might see/hear their ads everywhere. Thats because theyre spending millions to make sure youve heard of them.

Despite the great marketing, some of those companies have subpar services at best. Theyre more worried about making a sale than they are with actually delivering a quality product.

To be clear, a popular company with great marketing does NOT necessarily mean that they have a bad service. A couple of them have really great services! But you shouldnt assume that they have a great service just because they appear to be popular, and you also shouldnt write off lesser-known companiessome of these are new up-and-coming services who will eventually rise to the top of the market. They give you a unique opportunity to be along for the ride.

Mistake #2: Dont buy the cheapest OR the most expensive genetic test you can find.

The old mantra You get what you pay for applies here. However, price is a tricky quality to navigate.

On the one hand, you dont want something too cheap. A cheap Ancestry DNA test is most likely not the best dna test and will likely give you very little information. These tests will tell you things you already know about yourself, like which continent your genes came from. Sometimes cheap tests are simply trying to undercut the marketThey may be selling at a loss up front with the hopes that customers will buy more from them later.

On the other hand, you dont want to get ripped off by an over-priced DNA test. Expensive DNA tests may have a great product, but you can often find a product of similar (or even better) quality at a cheaper price.

You have to strike a comfortable middle ground. In our experience, roughly $100 is a fair price for a quality DNA test (give or take a few dollars). Aiming for a test around this amount will help ensure that you get a good product without over-paying.

Mistake #3: Dont confuse Accuracy with Precision.

Almost every DNA test company on the market claims to be the most accurate. Theyre not lying. DNA tests are typically 99.9% accurate. However, theyre often not precise.

Whats the difference between Accuracy and Precision?

For something to be accurate, it just needs to be true. If you have European heritage and your Ancestry DNA test comes back with results that simply say European, then its an accurate test. Its giving you results that are true, even if theyre not detailed.

For something to be precise, it has to be an exact expression of details. The most precise DNA tests currently on the market have at least 20 unique regions they use in their Ancestry reports. The best companies will have multiple regions on each continent in their reports (rather than having most of their tested regions all on the same continent).

However, you have to be wary of companies overselling how precise their tests are. Some companies claim to have hundreds of regions in their reports. In our experience, this is bending the truth a bit. Most of them really test for 20-30 regions, but then list the names of countries that are contained within those regions without actually testing DNA for them.

For example, if a DNA test determines that someone has Iberian Ancestry, one of these companies might list Spain and Portugal underneath and count those as 2 regions for marketing purposes even though they dont give a percentage breakdown for how much Iberian Ancestry is Spanish or Portugese.

In other words, some companies can be a little misleading with their marketing.

Our Top 3 Recommended DNA Tests

Now that weve shown you what to look out for, we want to share with you some of the best Ancestry DNA tests weve seen for discovering your heritage. .

We ranked the services by these 10 factors: 1) Company Reputation 2) Services Offered 3) Testing Method 4) Software Grade 5) Research & Scientific Evidence 6) CLIA Compliance 7) Customer Reviews 8) Price 9) Customer Service 10) Return Policy

Our Top Choice

CRI Genetics stands out as the best DNA Test for Ancestry for a few specific reasons. First, theyre headed by renowned genetic scientist with a reputation for leading exceptional studies in genetic science. While most genetic testing services rely on other peoples past research to produce their ancestry reports, CRI Genetics relies on someone who is currently doing Genome research.

Company Reputation:

CRI Genetics is led by Alexei Fedorov, Ph.D., who was mentored by Nobel Prize winning scientists at Harvard University and has gone on to spearhead many genetic studies of his own. As a company, CRI Genetics has established themselves as one of the top players in quality of service. They are the only DNA testing company we have come across that has any sort of money-back guarantee.

Details/Accuracy of Reports:

CRI Genetics currently offers 5 unique ancestry reports that are generated using a patented DNA analysis algorithm created by Alexei Fedorov. From a basic geographical breakdown of your Ancestry to a detailed history of your maternal or paternal line to an interactive Ancestry Timeline that pinpoints the year that certain heritages entered your family, the overall level of detail across all CRI Genetics reports is unmatched.See Full Report Here

#2 Choice

Company Reputation:Family Tree DNA was founded in the year 2000 by Bennett Greenspan, a businessman who was trying to solve mysteries within his own family history. The first tests offered to customers were very simple compared to todays DNA tests, but were considered advanced at the time.

Details/Accuracy of Reports:Today, Family Tree DNA offers a small range of reports with an above average level of detail. Their biggest strength is a very large database of customers, which helps with accuracy. View Full Report

#3 Choice

Company Reputation:Living DNA is a fairly young company, but have quickly risen in the ranks of DNA Testing companies with a vast network of connections with DNA experts. One thing is clear to us here at Genetics Digest: Living DNA is loved by their customers.

Details/Accuracy of Reports:Living DNAs Ancestry Reports have details for 80+ regions worldwide, but 21 of those regions are in Ireland and the United Kingdom. If you have a lot of British or Irish DNA, then this is definitely an interesting service for you to try. However, if your Ancestry is anything else, Living DNA is on par with most other services. View Full Report

Link:
Free Review of Ancestry Dna Tests | Genetics Digest

Recommendation and review posted by Bethany Smith

Molecular pathology services, including genetic testing, are rapidly becoming the standard of care in diagnostic medicine and other related areas. OHCA is committed to ongoing evaluation of the clinical evidence supporting the use of these services to ensure that medically necessary tests and technologies are available to our members.

On the OHCA proposed rule changes page, there is a sign up button for Web Alerts. These Web Alerts will send an email notification when there is a new posting for a proposed rule change. With each posting on this page, there is an opportunity to complete an electronic feedback form.

The OHCA seeks advice and consultation from medical professionals, professional and tribal organizations, and the general public in developing new or amended policies and rules. The proposed rule changes page is designed to give all constituents an opportunity to review and make comments regarding upcoming rule changes.

Disclaimer: The OHCA rules found on this Web site are unofficial. The official rules are published by the Oklahoma Secretary of State Office of Administrative Rules as Title 317 of the Oklahoma Administrative Code. To order an official copy of these rules, contact the Office of Administrative Rules at (405) 521-4911.

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OHCA - Genetic Testing

Recommendation and review posted by Bethany Smith

Pros and Cons of Genetic Testing: The human body is composed of millions of cells, which are considered as the basic units of life. Inside each cell lies the genetic material or the DNA (Deoxyribonucleic Acid).

Short sections of DNA are called together as the gene. The gene is also dubbed as the basic unit of heredity as it contains the information and instructions that dictate how the body should develop and function. Also, the gene is also important in the expression of inheritable characters and traits.

Previously weve seen disadvantages of genetically modified foods and genetic engineering pros & cons. In this article, well explore the pros and cons of genetic testing.

Genetic testing is a type of health program that involves the identification of any changes in genes, chromosomes, and proteins.

Do you have a family history of acquiring a specific disease? Or are you planning to have a child but afraid that he/she might inherit a trait you wouldnt want to? Genetic testing is the solution to all of these questions. The results of a genetic test confirm and eliminate the possibility of any suspected genetic disorder. Such results will be highly advantageous for the early treatment and prevention of diseases.

There are a lot of types of genetic testing depending on what you want to test. Genetic testing can range from biochemical tests, molecular approach, or simply family history questionnaires. To perform a genetic test, a tissue from any organ that usually develops during pregnancy can be obtained. Examples of such are the placenta, amniotic fluid (pregnant womans water), bone marrow, or blood.

Now we will explore the pros and cons of genetic testing. First, lets focus on pros.

There are a lot of potential advantages which can arise as a result of genetic testing. The following are some of them.

As with any disease, early diagnosis of the disease will greatly help in faster treatment. The results of genetic testing can also help your healthcare provider in predicting the likelihood and deciding about the management of the disorder. In addition, the results of the test can also help one to learn more about the genetic disease and how it may possibly affect them and their relatives as well.

For some people, finding out that they do not have the gene for a certain disease can become a blessing. They may feel a lot more peace because of the fact that they have not passed any gene abnormality to their children. In addition, because they no longer require the same type of medical treatment as with people who have the gene, the resources can be allotted to those who have the risk of having the disease.

Genetic tests can be helpful in establishing evidence for the parenthood of a person for a case like child custody and support. The results of genetic test can also be used as a support for placing a parents name on the birth certificate of a child. Depending upon the country/state where you live in, DNA testing can be ordered by the judge for settling disputes in child custody laws.

For instance, if there is a low probability of passing a certain unwanted genetic condition, couples can have be assured that they can have children free of the disorder. On the other hand, a positive result may give the couple an idea of deciding not to have children because doing so may result to a high risk of their child developing the condition.

Like how it can determine parenthood, being genetically tested can be helpful is determining and interpreting developmental delays in children. Reasons for significant lags in physical, mental, and emotional growth can be determined.

Also if a woman has two or more miscarriages or pregnancy over age 34, genetic testing will be helpful for early diagnosis which can help identify the appropriate treatment options.

While the process has great advantages indeed, there are several disadvantages that a person who wishes to undergo testing should be aware of. The following are some of them.

The physical risks associated with most genetic tests are indeed very small as some tests only require mere blood or tissue samples. However, some tests can be really destructive. As an example, the methods for prenatal testing involves the acquisition of amniotic fluid around the fetus. Such practice can be really dangerous because the mother may suffer from miscarriage.

As alluded to earlier, the results of genetic testing can provide freedom from any uncertainty. However, in some cases, the results of genetic testing may create an emotional trauma for the person who finds out that he/she has a certain disease. It can lead to an increased anxiety to the individual as he might blame himself for possessing a gene that causes the disorder and potentially passing it onto their children.

About this, the results of these tests may also create tension among family members when information about a family member is revealed. Having a negative test can cause emotional distress to the person because it gives him/her the feeling of survivor guilt from being unaffected by the disease while his/her sibling is at risk.

Genetic discrimination is the condition wherein a person feels and gets discriminated due to the fact that he/she possesses a genetic abnormality that increases the chances of him/her developing a certain genetic disorder. And because the results of genetic tests are included in a persons medical history, the fact that he/she has this abnormality becomes known to employers and other people in the workplace. As a result, people may treat him/her differently.

While it is true that some tests can be very specific about the genetic disorder, these test often cannot tell the severity of the manifestation of the disease. Also, a negative result may not be conclusive because it is not possible for a single test to identify all the genetic changes and abnormalities in a certain disorder. Because of this, additional tests may be necessary. Another thing is that while most genetic disorders can be easily diagnosed using these tests, there are still potentially millions of genetic mutations which are still not understood. Furthermore, treatment strategies are still lacking.

For instance, one disadvantage of using biochemical test as a genetic test is that proteins from the tissue samples are more unstable that the gene itself. Easy deterioration of samples means a higher chances of inaccuracy in the results. Therefore, they should be properly stored and analyzed immediately after obtaining.

Basically, the price of having genetic test will depend on various factors including the type of test and the clinic you visit. According to the National Human Genome Research Institute, the average cost of genetic testing in the US can range from less than $100 to $2,000! And as mentioned above, a single test may not be able to determine all genetic abnormalities so additional tests may be advised. The expensive price of genetic testing is only suitable for a small groups of patients because only those who can afford it can be tested.

It is important to note that not all tests have the same predictability. The accuracy of any result would be of course depend on whether the disorder is caused by an abnormality of the gene and chromosome or just a mere result of acquisition from the environment.

According to a study by the Harvard School of Public Health, a large majority of Americans are not into adopting this kind of genetic technology. In fact, only 6 percent of adults said that they had undergone genetic testing. While genetic testing is not compulsory, just like any medical intervention, this technique aims to do good than to harm.

However, some consequences of the process are inevitable. Therefore, to avoid such complications, it is vital to have counseling before and after genetic testing. In this way, individuals are free to choose whether they want to or dont want to undergo testing. And if needed, they could have extra support.

So if youre planning to be genetically tested, you might want to ponder about this question: Is having genetic testing a mere trend that offers unproven hope, or does it represent the first sign of treatment for affected patients? What do you think?

12 Pros and Cons of Genetic Testing

Read more:
12 Pros and Cons of Genetic Testing | Biology Explorer

Recommendation and review posted by Bethany Smith

Human life is dependent upon the hearts ability to pump forcefully and frequently enough, but heart failure signs can disturb its normal function. Most humans cannot live more than four minutes without a heartbeat or continuous blood-flow. At that time, brain cells begin to die because they lack adequately oxygenated blood-flow.

The human adult body requires, on average, 5.0 liters of re-circulated blood per minute. In the cardiology field, this metric is called the Cardiac Output, which is calculated as Stroke Volume (SV) x Heart Rate (HR). Another key metric is a patients Ejection-Fraction (EF %). A patients EF tells a cardiologist and other physicians if his or her heart is functioning normally or low normally. It is a measurement of ones heart contraction, with a normal EF range being 55-70%.

This number can also be combined with a patients heart rate to provide physicians with a baseline of a patients cardiac status. A normal range for an adult is 60-100 beats per minute, and this can be significantly higher during a normal pregnancy.

In this article:

For a cardiologist, cardiac metrics indicate if their services are required and allowthem to sign-off on pre-operative cardiac clearances. For other physicians, it tells them if the organ which they specialize in is being perfused adequately (for example, a nephrologist would be interested to know kidney perfusion). It can also indicate the degree to which decreased heart function may affect the severity or spread of disease.

When the heart fails to contract forcefully enough and its performance decreases to the point where its ability to circulate blood adequately is compromised (the EF% falls below 40%), this is considered heart failure. The clinical parameters of heart failure are clearly defined by the New York Heart Association (NYHA), which places patients in NYHA Class III & IV into the heart failure category.

An echocardiogram (often called an Echo), as opposed to an Electrocardiogram (EKG or ECG), allows technicians and physicians to visualize the beating heart. Video clips of the heart contracting are digitally recorded, and a patients EF and Cardiac Output (CO) can be measured with several diagnostic tools (Fractional Shortening via 2D or M-Mode measurements and Simpsons Method via 2D and 3D Quantification) on a cardiovascular ultrasound system.

When an experienced echo tech or cardiologist views a failing heart, it is immediately apparent. Based on my experience reading echocardiograms, I can see that the heart walls or heart muscles (myocardium) are not contracting as vigorously as they should.

For patients with a 5% EF range, any physical movement is extremely strenuous, and they can go into cardiac arrest at any moment, which is why they are usually on cardiac telemetry in a hospital setting. Most likely, a patient with 5% EF range would be awaiting a heart transplant, unless there is a medical condition preventing them from being eligible.

Once a patient falls into the heart failure range, they will be lethargic and have severe limits on activities. Other clinical manifestations of heart failure can include peripheral edema (i.e. swelling in the feet, legs, ankles, or stomach), pulmonary edema, and shortness of breath. In many cases, this can lead to depression.

In evaluating the frequency of heart failure in the U.S, statistics from the U.S. Centers for Disease Control (CDC) find that approximately 5.7 million adults are afflicted with this condition. Additionally, care for congestive heart failure costs an estimated $30.7B per year. Furthermore, the mortality rates of patients suffering from heart failure indicate its clinical severity, with 1 in 5 patients with this condition dying within a year of receiving the diagnosis.

A patient experiencing severe heart failure has limited treatment options, which are expensive, complicated, and have major lifestyle implications.

These limited options include:

Consequently, physicians need more effective weapons for treating heart failure in order to improve patients lives and reduce healthcare-related costs. CHF patients have disproportionate hospital readmission rates when compared to other major diseases.

Enter in the growing field of cardiac stem cell treatments, which introduce fundamentally new treatment options for heart failure patients. In cardiac stem cell treatments, stem cells are taken from a patients bone marrow or fat tissue in a sterile surgical procedure and injected via a catheter-wire into infarcted or poorly contracting muscular segments of the hearts main pumping chamber, the left ventricle (LV).

Over the course of a few months, the stem cells impact myocardial cells and begin to improve the contractility of the affected segments, most likely through paracrine signaling mechanisms and impacting the local microenvironment. This can bring a patients EF to low-normal or even normal levels. As a result, a patient can live a more normal life and return to many activities.

A very early clinical trial aimed at evaluating the potential and effectiveness of cardiac stem cell therapy in humans was conducted in 2006 utilizing a commercial product, VesCellTM. The parameters and results of this trial were documented in the American Heart Associations Circulation, Abstract 3682: Treatment of Patients with Severe Angina Pectoris Using Intracoronarily Injected Autologous Blood-Borne Angiogenic Cell Precursors.The subjects of this trial received an intracoronary injection of VesCellTM, an Autologous Angiogenic Cell Precursor (ACP)-based product.

The authors drew their conclusion regarding this study. VesCell therapy for chronic stable angina seems to be safe and improves anginal symptoms at 3 and 6 months. Larger studies are being initiated to evaluate the benefit of VesCell for the treatment of this and additional severe heart diseases. (Source: Tresukosol et al. Abstract 3682: Treatment of Patients with Severe Angina Pectoris Using Intracoronarily Injected Autologous Blood-Borne Angiogenic Cell Precursors. Circulation. October 31, 2006. Vol. 114, Issue Suppl 18. Link: http://circ.ahajournals.org/content/114/Suppl_18/II_786.4 )

Another early cardiac stem cell clinical trial was performed in 2009 by a Cedars-Sinai team based on technologies and discoveries made by Eduardo Marban, MD, PhD, and led by Raj Makkar, MD. In this study, they explored the safety of harvesting, expanding, and administering a patients cardiac stem cells to repair heart tissue injured by myocardial infarction.

Recently, the American College of Cardiology (ACC) also announced results of a ground-breaking clinical study to evaluate the efficacy and effectiveness of cardiac stem cell treatment for heart failure patients. As stated by Timothy Henry, M.D., Director of Cardiology at Cedars-Sinai Heart Institute and one of the studys lead authors, This is the largest double-blind, placebo-controlled stem cell trial for treatment of heart failure to be presentedBased on these positive results, we are encouraged that this is an attractive potential therapy for patients with class III and class IV heart failure.

Additionally, Dr. Charles Goldthwaite, Jr, published a whitepaper titled, Mending a Broken Heart: Stem Cells and Cardiac Repair, in which he draws the conclusion, Given the worldwide prevalence of cardiac dysfunction and the limited availability of tissue for cardiac transplantation, stem cells could ultimately fulfill a large-scale unmet clinical need and improve the quality of life for millions of people with CVD. However, the use of these cells in this setting is currently in its infancymuch remains to be learned about the mechanisms by which stem cells repair and regenerate myocardium, the optimal cell types, and modes of their delivery, and the safety issues that will accompany their use.

Clearly, there is a trend toward acceptance of cardiac stem cell therapies as an emerging treatment option. Several world-renowned institutes are now conducting clinical studies involving cardiac stem cell treatment, as well as applying for intellectual property protection (patents) pertaining to the techniques required in administrating the therapies.

The key questions at this point in time appear to be:

An important whitepaper pertaining to cardiac stem cells is Ischemic Cardiomyopathy Patients Treated with Autologous Angiogenic and Cardio-Regenerative Progenitor Cells, written by Dr. Athina Kyritsis, et al. In it, the physicians describe their objective as investigating the feasibility, safety, and clinical outcome of patients with Ischemic Cardiomyopathy treated with Autologous Angiogenic and Cardio-Regenerative Progenitor cells (ACPs).

The researchers state: In numerous human trials there is evidence of improvement in the ejection fractions of Cardiomyopathy patients treated with ACPs. Animal experiments not only show improvement in cardiac function, but also engraftment and differentiation of ACPs into cardiomyocytes, as well as neo-vascularization in infarcted myocardium. In our clinical experience, the process has shown to be safe as well as effective.

The authors also found that patients treated with this approach gained increases in cardiac ejection fraction from their starting measurements, with improvements in their cardiac ejection fraction of 21 points (75% increase) at rest and 28.5 points (80% increase) at stress. As a result of these finding, the authors conclude, ACPs can improve the ejection fraction in patients with severely reduced cardiac function with benefits sustained to six months.

In the practice of medicine, the focus should be on delivering excellent care to patients. If there are cardiac stem cell treatments available, then regulatory obstacles should be removed when sufficient clinical trial evidence has been provided to indicate safety and efficacy.

Cardiologist Zannos Grekos, MD, a pioneer in cardiac stem cell therapy since 2006, points to the vastly untapped promise of related therapies, commenting Those of us that have been involved with cardiac stem cell treatment for the last 10-plus years can see the incredible potential this approach has.

As of 2017, the U.S. healthcare system is under enormous pressure to deliver affordable healthcareto a growing population of patients, especially those who are fully or partially covered under Medicare or Medicaid (many have secondary coverage). Although we are in the infancy of its development, cardiac stem cell treatments represent a potentially powerful treatment alternative to patients with heart failure symptoms.

To learn more, view the resources below.

1) Regenocyte http://www.regenocyte.com

2) Cleveland Clinic Stem Cell Therapy for Heart Disease my.clevelandclinic.org/health/articles/stem-cell-therapy-heart-disease

3) Harvard Stem Cell Institute (HSCI) hsci.harvard.edu/heart-disease-0

4) Cedars Sinai Cardiac Stem Cell Treatment http://www.cedars-sinai.edu/Patients/Programs-and-Services/Heart-Institute/Clinical-Trials/Cardiac-Stem-Cell-Research.aspx

5) Johns Hopkins Medicine Cardiac Stem Cell Treatments http://www.hopkinsmedicine.org/stem_cell_research/cell_therapy/a_new_path_for_cardiac_stem_cells.html

What do you think about heart failure signs and cardiac stem cell therapies? Share your thoughts in the comments section below.

Up Next:European Society of Cardiology (ESC) Congress Presentation Reveals Results From Pre-Clinical Study Using CardioCells Stem Cells for Acute Myocardial Infarction

Guest Post: This is a guest article by Clifford M. Thornton, a Certified Cardiovascular Technologist, experienced Echocardiographer Technician, and journalist in the cardiac and medical device fields. His articles have been published in Inventors Digest, Global Innovation Magazine, and Modern Health Talk. He is enthusiastic about progress with cardiac stem cell therapies and their role in heart failure treatment.He can be reached byphone at 267-524-7144 or by email at[emailprotected].

Editors Note This post was originally published on March 14, 2017, and has been updated for quality and relevancy.

Heart Failure Signs | Cardiac Stem Cell Therapies for Heart Failure Treatment

Excerpt from:
Heart Failure Signs | Cardiac Stem Cell Therapies: Heart ...

Recommendation and review posted by Bethany Smith

The U.S. Food and Drug Administration (FDA) has granted AVXS-101 Orphan Drug Designation for the treatment of all types of SMA and Breakthrough Therapy Designation, as well as Fast Track Designation, for the treatment of SMA Type 1.

The European Medicines Agency (EMA) also granted AveXis access into its PRIority Medicines (PRIME) program for AVXS-101 for the treatment of SMA Type 1.

The open-label, single-arm, single-dose, multi-center trial known as STR1VE is designed to evaluate the efficacy and safety of a one-time IV infusion of AVXS-101 in patients with SMA Type 1. The co-primary efficacy outcome measures of the trial include the achievement of independent sitting for at least 30 seconds at 18 months of age; and, event-free survival at 14 months of age. Co-secondary outcome measures include the ability to thrive, and the ability to remain independent of ventilatory support at 18 months of age.

The open-label, dose-comparison, multi-center Phase 1 trial known as STRONG is designed to evaluate the safety, optimal dosing, and proof of concept for efficacy of AVXS-101 in two distinct age groups of patients with SMA Type 2, utilizing a one-time IT route of administration. The primary outcome measure for patients less than 24 months of age at the time of dosing is the achievement of the ability to stand without support for at least three seconds. The primary outcome measure for patients between 24 months and 60 months of age at the time of dosing is the achievement of change in Hammersmith Functional Motor Scale Expanded from baseline. The secondary outcome measure for both age groups is the proportion of patients that achieve the ability to walk without assistance, defined as taking at least five steps independently while displaying coordination and balance. Developmental abilities, including motor function, will also be evaluated as exploratory objectives.

Learn more about clinical trials

We have exclusive worldwide license agreements to develop and commercialize gene therapy using the AAV9 vector to treat two rare neurological monogenic disorders: Rett syndrome (RTT) and a genetic form of amyotrophic lateral sclerosis (ALS) caused by mutations in the superoxide dismutase 1 (SOD1) gene.

Read the rest here:
AveXis Research & Development

Recommendation and review posted by Bethany Smith

Stem cells are biological cells that are able to stay dormant until triggered to reproduce into new tissue. Found in human embryos and in adult tissue, they can form into any cell type, and help repair organs and skin in the case of injury or other cause of damage.

So is it any surprise that their potential is also being trumpeted in the world of skin care? Cosmetic science has often taken inspiration from hard-core medical breakthroughs, and stem cells appear to possess the ideal skill set to throw the switch on a veritable fountain of youth.

While skin stem cells have found use in treating diseases, stem cells technology in skin care products have been largely based on hype rather than science, but in some cases like RG-CELL, it truly works magic.

The concept of topically applying stem cells, through cream, serum, mask, or facial procedure, with a promise to replenish dying cells and regenerate dying tissues has shown no real scientific evidence that it works.

If youre unfamiliar with the practice, you may question the validity of using live stem cells in anti-aging products when its already an enormous and time consuming challenge to use them in actual organ regenerating procedures.

Firstly, stem cells are highly unstable. They have little to no shelf life. Secondly, they will not enter the deep layers of the skin without an effective skin delivery system. And thirdly, stem cells need specific nutrition via a blood supply in the tissue to survive and function if they were layered onto intact skin the stem cells would just die.

It should be made abundantly clear that, no stem cell skin care products contain actual stem cells. Stem cell based products contain growth factors, along with enzymes and other nutrients, which help the cells grow. Other products dont contain any stem cell-related material at all.

[frame src=https://rg-cell.com/wp-content/uploads/2013/05/stem-cell-skin-care.jpg width=250 height=188 alt=Stem Cell Skin Care align=right]There are 2 ways in which stem cell technology is being used. Firstly, companies are creating products with specialized peptides and enzymes or plant growth factors which, when applied topically on the surface, help protect the human skin from damage and deterioration. Products claiming to contain plant stem cells dont contain human cytokines (or cell messengers), and in fact are really just ground up plant bits. In short, plant stem cell technology cannot effectively impact human stem cells. It can be useful as excellent antioxidants, but marketing has made the benefits bigger than reality.

Secondly, and bearing more scientific evidence, is an alternative application of skin care anti-aging products. These products utilize human stem cell technology, and your skin is the most active participant, NOT plant or apple stem cells. Using ingredients that promote the repair and rejuvenation of your skin by stimulating the activity of your own stem cells in the skin has proven to be safer, more ethical and far more scientifically proven than applying stem cells in a jar. This technology implies a superior product designed specifically to regenerate and rejuvenate your own skin cells.

These products contain epidermal growth factors (EGF) obtained by genetic engineering technology (microbial recombinant) totally identical to natural EGF, known as a BEAUTY FACTOR, boosts and regulates stem cell proliferation. When applied to the skin, stimulate collagen production, improve elasticity, firm sagging skin, improve tone and so much more.

[frame src=https://rg-cell.com/wp-content/uploads/2012/11/nano-encapsulation.jpg width=250 height=190 alt=Skin Delivery System align=right]EGF is a large molecule so it cannot penetrate the skin. In fact, it is too big to fit in between the spaces in cells of our skin. There is also speculation around the length of time, that it can remain stable in a formulation. Clinical studies and research are practically non-existent. Therefore, buyer beware: If you opt for using a product that contains EGF consider whether or not the mechanism of action employed to deliver the ingredient to the dermal layers, will actually work.

Only special technology, can deliver EGF into the skin deeper layers. One of the biggest advances is the use of a patented nano-particulate lipid bi-layer delivery system that allows the products to be delivered deep into the skin where the stem cells live.

RG-Cell uses a unique patented nano-encapsulation technology as its delivery system. This improves the permeation and penetration efficiency of the active ingredients. Owing to this fact, RG-CELL can make valid claims about the efficiency in it is delivery of EGF where it is needed the most. This technology also stabilizes the EGF thereby prolonging its shelf life in the actual product.

Thus we can see that there are already many choices in skin care products with specialized peptides and enzymes or EGFs which, when applied topically stimulate the skins own stem cells. But, only one uses the most advanced technology to deliver nutrients into the skin. Expect many more good choices to be developed in the years to come!

Go here to read the rest:
Stem Cells Used in Anti-Aging Skin Care Radiant RG-Cell

Recommendation and review posted by Bethany Smith

CRISPR is a term used in microbiology. It stands for Clustered Regularly-Interspaced Short Palindromic Repeats. These are a natural segment of the genetic code found in prokaryotes: most bacteria and archaea have it.[1]

CRISPR has a lot of short repeated sequences. These sequences are part of an adaptive immune system for prokaryotes. It allows them to remember and counter other organisms that prey on them, such as bacteriophages.

They have the potential to modify the genes of almost any organism. They are part of a tool that allows precisely targeted cutting and insertion of genes in genetic modification (GM). Work is under way to find how they can be used to attack virus diseases in humans.[2]

Each repetition is followed by short segments of "spacer DNA" from previous exposures to a bacterial virus or plasmid.[2] CRISPR spacers recognize and cut up the foreign genetic elements in a manner like RNA interference in eukaryotic organisms.

In effect, the spacers are fragments of DNA from viruses that have previously tried to attack the cell line. The foreign source of the spacers was a sign to researchers that the CRISPR/cas system could have a role in adaptive immunity in bacteria.[3]

The actual cutting is done by a nuclease called Cas9. Cas9 has two active cutting sites, one for each strand of the DNA's double helix. Cas9 does this by unwinding foreign DNA and checking whether it is complementary to the 20 basepair spacer region of the guide RNA (the spacer region RNA). If it is, the foreign DNA gets chopped up.

The technology has been used to switch off genes in human cell lines and cells, to study Candida albicans, to modify yeasts used to make biofuel and to genetically modify crop strains.[4]

See original here:
CRISPR - Simple English Wikipedia, the free encyclopedia

Recommendation and review posted by Bethany Smith

Posted on September 11th, 2018 by Dr. Francis Collins

Caption: A CRISPR/cas9 gene editing-based treatment restored production of dystrophin proteins (green) in the diaphragm muscles of dogs with Duchenne muscular dystrophy.Credit: UT Southwestern

CRISPR and other gene editing tools hold great promise for curing a wide range of devastating conditions caused by misspellings in DNA. Among the many looking to gene editing with hope are kids with Duchenne muscular dystrophy (DMD), an uncommon and tragically fatal genetic disease in which their musclesincluding skeletal muscles, the heart, and the main muscle used for breathinggradually become too weak to function. Such hopes were recently buoyed by a new study that showed infusion of the CRISPR/Cas9 gene editing system could halt disease progression in a dog model of DMD.

As seen in the micrographs above, NIH-funded researchers were able to use the CRISPR/Cas9 editing system to restore production of a critical protein, called dystrophin, by up to 92 percent in the muscle tissue of affected dogs. While more study is needed before clinical trials could begin in humans, this is very exciting news, especially when one considers that boosting dystrophin levels by as little as 15 percent may be enough to provide significant benefit for kids with DMD.

Posted In: News

Tags: animal models, beagles, Cavalier King Charles Spaniel, CRISPR, CRISPR/Cas9, diaphragm muscle, DMD, dogs, Duchenne muscular dystrophy, dystrophin, gene editing, genetic diseases, heart, muscle, muscular dystrophy, rare diseases, Somatic Cell Genome Editing

Posted on October 10th, 2017 by Dr. Francis Collins

About a month ago, I had the pleasure of welcoming the Juip (pronounced Yipe) family from Michigan to NIH. Although youd never guess it from this photo, two of the Juips five children9-year-old Claire and 11-year-old Jake (both to my left)have a rare genetic disease called Friedreichs ataxia (FA). This inherited condition causes progressive damage to their nervous systems and their hearts. No treatment currently exists for kids like Claire and Jake, yet this remarkable family has turned this serious health challenge into an opportunity to raise awareness about the need for biomedical research.

One thing that helps keep the Juips optimistic is the therapeutic potential of CRISPR/Cas9, an innovative gene editing systemthat may someday make it possible to correct the genetic mutations responsible for FA and many other conditions. So, Im sure the Juips were among those encouraged by the recent news that NIH-funded researchers have developed a highly versatile approach to CRISPR/Cas9-based therapies. Instead of relying on viruses to carry the gene-editing system into cells, the new approach uses tiny particles of gold as the delivery system!

Posted In: Health, Science, technology, Uncategorized

Tags: CRISPR, CRISPR-Gold, CRISPR/Cas9, DMD, Duchenne muscular dystrophy, dystrophin, FA, Friedreichs ataxia, gene editing, Juip, rare diseases, stem cells

Posted on July 18th, 2017 by Dr. Francis Collins

Credit: Seth Shipman, Harvard Medical School, Boston

Theres a reason why our cells store all of their genetic information as DNA. This remarkable molecule is unsurpassed for storing lots of data in an exceedingly small space. In fact, some have speculated that, if encoded in DNA, all of the data ever generated by humans could fit in a room about the size of a two-car garage and, if that room happens to be climate controlled, the data would remain intact for hundreds of thousands of years! [1]

Scientists have already explored whether synthetic DNA molecules on a chip might prove useful for archiving vast amounts of digital information. Now, an NIH-funded team of researchers is taking DNAs information storage capabilities in another intriguing direction. Theyve devised their own code to record information not on a DNA chip, but in the DNA of living cells. Already, the team has used bacterial cells to store the data needed to outline the shape of a human hand, as well the data necessary to reproduce five frames from a famous vintage film of a horse galloping (see above).

But the researchers ultimate goal isnt to make drawings or movies. They envision one day using DNA as a type of molecular recorder that will continuously monitor events taking place within a cell, providing potentially unprecedented looks at how cells function in both health and disease.

Posted In: Health, Science, Video

Tags: biosensor, biotechnology, Cas1, Cas2, CRISPR, CRISPR-Cas, DNA, DNA movie, DNA storage, E. coli, film, gene editing, genomics, Human and Animal Locomotion, imaging, information storage, molecular recorder, movie, spacers

Posted on May 4th, 2017 by Dr. Francis Collins

Jesse Dixon

As a kid, Jesse Dixon often listened to his parents at the dinner table discussing how to run experiments and their own research laboratories. His father Jack is an internationally renowned biochemist and the former vice president and chief scientific officer of the Howard Hughes Medical Institute. His mother Claudia Kent Dixon, now retired, did groundbreaking work in the study of lipid molecules that serve as the building blocks of cell membranes.

So, when Jesse Dixon set out to pursue a career, he followed in his parents footsteps and chose science. But Dixon, a researcher at the Salk Institute, La Jolla, CA, has charted a different research path by studying genomics, with a focus on understanding chromosomal structure. Dixon has now received a 2016 NIH Directors Early Independence Award to study the three-dimensional organization of the genome, and how changes in its structure might contribute to diseases such as cancer or even to physical differences among people.

Posted In: Health, Science

Tags: 2016 NIH Directors Early Independence Award, 3D genome structure, chromatin, chromatin structure, CRISPR, CRISPR/Cas9, DNA, DNA packaging, ENCODE, Encyclopedia of DNA Elements, enhancer, gene editing, genome, genomics, histones, TAD, topologically associated domains

Posted on January 24th, 2017 by Dr. Francis Collins

Caption: This image represents an infection-fighting cell called a neutrophil. In this artists rendering, the cells DNA is being edited to help restore its ability to fight bacterial invaders.Credit: NIAID, NIH

For gene therapy research, the perennial challenge has been devising a reliable way to insert safely a working copy of a gene into relevant cells that can take over for a faulty one. But with the recent discovery of powerful gene editing tools, the landscape of opportunity is starting to change. Instead of threading the needle through the cell membrane with a bulky gene, researchers are starting to design ways to apply these tools in the nucleusto edit out the disease-causing error in a gene and allow it to work correctly.

While the research is just getting under way, progress is already being made for a rare inherited immunodeficiency called chronic granulomatous disease (CGD). As published recently in Science Translational Medicine, a team of NIH researchers has shown with the help of the latest CRISPR/Cas9 gene-editing tools, they can correct a mutation in human blood-forming adult stem cells that triggers a common form of CGD. Whats more, they can do it without introducing any new and potentially disease-causing errors to the surrounding DNA sequence [1].

When those edited human cells were transplanted into mice, the cells correctly took up residence in the bone marrow and began producing fully functional white blood cells. The corrected cells persisted in the animals bone marrow and bloodstream for up to five months, providing proof of principle that this lifelong genetic condition and others like it could one day be cured without the risks and limitations of our current treatments.

Posted In: Health, Science

Tags: adult stem cells, bacteria, CGD, chronic granulomatous disease, clinical trials, CRISPR, CRISPR-Cas, CRISPR/Cas9, DNA editing, fungi, gene therapy, genetics, hematopoietic stem cells, immunodeficiency, immunology, infectious disease, inherited immuodeficiency, neutrophil, rare disease, translational medicine, X chromosome, X-linked chronic granulomatous disease

Originally posted here:
CRISPR NIH Director's Blog

Recommendation and review posted by Bethany Smith

What is Cell and Gene Therapy for Cancer?Gene therapy is a technique that uses genes to treat or prevent disease such as cancer by inserting a gene into a patients cells instead of using drugs or surgery. Researchers are testing several approaches to gene therapy, including: replacing a mutated or abnormal gene that causes disease with a healthy copy of the gene; inactivating, or knocking out, a mutated gene that is functioning improperly; and introducing a new gene into the body to help fight a disease.

Cell Therapy is the infusion or transplantation of whole cells into a patient for treatment of an inherited or acquired disease like cancer.

Primary Forms of Cell and Gene Therapies for Cancer Treatment

The long-term goal of cancer cell and gene therapy is to develop treatments that attack only cancer cells, eliminating adverse effects on the body. Furthermore, these therapies have potential for treating other diseases such as cardiovascular, disorders, cystic fibrosis, hemophilia, sickle-cell anemia, muscular dystrophy, diabetes, and Parkinsons. All research in this area, therefore, makes a difference.

About Molecular Medicine

Molecular medicine uses the bodys own cells and genes as both the source and medicine for diseases of all types the basis for all cell and gene therapies.

Molecular medicine began with the identification of DNA in the early 1900s. Progress was slow until the mapping of the human genome in the new millennium and the rapid technological advances that made it possible to isolate and target specific cells and genes.

This field of study explains the fundamental genetic errors that cause diseases like cancer and helps establish a blueprint for good health.

Molecular medicine and advanced technology make it possible to target cancers directly without damage to other parts of the body.

Molecular medicine is also referred to as genetic medicine, gene therapy, targeted therapeutics, genetic epidemiology or individualized medicine.

The rest is here:
Cell and Gene Therapy | Alliance for Cancer Gene Therapy ...

Recommendation and review posted by Bethany Smith

Industry Outlook

The global gene therapy market sizewas valued at USD 7.6 million in 2017. It is estimated to expand at a CAGR of over 19.0% during the forecast period. Increasing number of molecules in the development phase is expected to stoke market growth. It was projected that in 2016, more than 900 molecules are in the development phase that can prove to be an effective treatment for several incurable diseases, which are generally caused by an error in a single gene.

Increasing gene therapy innovations for cardiovascular and rare diseases treatment is one of the key trends driving the market. Rising focus on development of gene therapy treatment for rare diseases is a result of intensifying competition among market players to consolidate their position in the industry.

Gene therapy involves incorporation of an artificial or a modified gene using modified viral vectors that help deliver the gene at intended site of action or even kill the cell that may cause the disease. This treatment is mostly a one-time treatment or requires very few doses of medication to completely cure the disease.

The method of treatment, which was once considered impossible, has now become a trend among big and small companies. A consequence of this has been an upsurge in the number of successful startups, backed by investors in line with big companies. The trends is poised to continue and boost the growth of the market during the forecast period.

Some novel molecules to be used for gene therapy are set to reach the commercialization stage. The growth of the market is largely dependent on key decisions made by manufacturers such as pricing, regulations, and reimbursement for treatment along with payers who help cover treatment costs. However, concerns regarding unethical use of the therapy can hamper growth prospects, especially in developing countries.

The gene therapy market is witnessing an upswing in innovations in various therapeutic fields of medicine. However, oncology is at the forefront in terms of innovation. On the basis of indication, the cancer segment accounted for the leading share of the overall market revenue in 2017. This is due to the high number of pipeline molecules that were registered over the last three years. Rising prevalence of cancer caused due to genetic mutations is also contributing to the growth of the segment. The genetic disorders segment, however, is anticipated to register the highest CAGR during the forecast period.

There are very few drugs in the market that have been approved by various regulatory bodies across the globe. These drugs are considered to change the treatment methods and regimen for rare and orphan diseases, however, their sky high pricing is limiting their commercial success.

For instance, Glybera, the first drug approved for the treatment of LPLD, was a breakthrough in the medical history. However, the drug couldnt be a commercial success due to high pricing at 1.6 million USD at the time of launch and very low prevalence of the disease (1-2 per a million of population).

In 2016, GlaxoSmithKline got another drug Strimvelis approved by European drug regulatory authority for the treatment of ADA-SCID. Other techniques in R&D are likely to witness a significant growth rate in the forecast years due to proven success of approved drugs.

The key element of gene therapy lies in the delivery of modified gene or functioning gene. Delivery systems used should be able to deliver functioning gene to intended cell target through modified viruses, which are considered the best vectors by scientists as viruses are highly evolved in delivering nucleic acid, bypassing the immune system of the host.

Several viruses such as Adeno-associated virus, retrovirus, lentivirus, and herpes simplex virus are modified in labs and are used as carriers for gene therapy drugs. Adenovirus is the most used viral vector followed by Retrovirus due to their reduced immunogenicity. Each of the viruses has its own disadvantage such as toxicity, limited DNA carrying capacity, etc.

Non-Viral vectors are being developed lately that can reduce or eliminate viral toxicity completely, however, none of the non-viral vectors possess ideal vector properties as of now.

Europehas seen two efficient gene therapy molecules after 2010, one was released in 2012 by UniQure N.V and other by GlaxoSmithKline, which were groundbreaking and were approved by the European regulatory body.

The U.S. is estimated to become a leader in terms of revenue as the country more than 64.0% of clinical trials by various big and small companies in the overall clinial trials. Among emerging economies, Russia and China are expected to be at the forefront of the market by a significant margin as they have two approved drugs in the market that can be used for cancer treatment.

Some of the key players are UniQure N.V, Spark Therapeutics LLC, Bluebird Bio, Juno Therapeutics, GlaxoSmithKline, Celgene Corporation, Shire Plc, Sangamo Biosciences, Dimension Therapeutics, Voyager Therapeutics, Human Stem Cell Institute, Bristol Myers Squibb, and Chiesi Farmaceutici S.p.A.

Due to a large number of pipeline molecules in development and intense competition among companies to augment their revenue growth, the market is projected to tread along a healthy growth track. Most of the startups are attracting capital investments to support their research for new molecules and initiate new product development.

Attribute

Details

Base year for estimation

2016

Actual estimates/Historical data

2014 - 2016

Forecast period

2017 - 2025

Market representation

Revenue in USD Million and CAGR from 2017 to 2025

Regional scope

North America, Europe, Asia Pacific, Latin America, Middle East & Africa

Country scope

U.S., Canada, Germany, U.K., China, Japan, Brazil, South Africa

Report coverage

Revenue forecast, company share, competitive landscape, growth factors and trends

15% free customization scope (equivalent to 5 analyst working days)

If you need specific information, which is not currently within the scope of the report, we will provide it to you as a part of customization

This report forecasts revenue growth and provides an analysis of themarket trends in each of the sub-markets from 2014 to 2026. For the purpose of this report, Grand View Research has segmented the global gene therapy market report on the basis of indication, vector type, and region:

Indication Outlook (Revenue, USD Million, 2014 - 2026)

Cancer

Cardio Vascular Diseases

Infectious Diseases

Genetic Disorders

Neuro Disorders

Others

Vector Type Outlook (Revenue, USD Million, 2014 - 2026)

Viral Vectors

Retrovirus

Adenovirus

Adeno-associated virus

Vaccinia virus

Herpes simplex virus

Others

Non-Viral Vectors

Injection of Naked DNA

Lipofection

Others

Regional Outlook (Revenue, USD Million, 2014 - 2026)

North America

Europe

Asia Pacific

Latin America

MEA

See more here:
Gene Therapy Market Share Insights - Grand View Research

Recommendation and review posted by Bethany Smith

SC21 BioTech: Stem Cell Skin Care Set

SC21 nowoffers a rejuvenating stem cell skin careset that is available to help restore aging skin. At SC21, we have been able to combine human mesenchymal stem cell growth factors, polypeptide complexes, and cytokines, with our day time anti-aging cream & evening hydration serum.

Our SC21 biotechnology scientists have developed a process to isolate potent rejuvenating factors from human stem cells. By resupplying the skin with these powerful missing factors, SC21 Day & Night Stem Cell Skin Care promotes cell renewal, boosts the production of collagen and elastin, restores aging cells, and, ultimately, provides you with more youthful looking skin.

It is important to note that as we age, the stem cell population that is vital in providing healing signals to the skin dramatically diminishes. As a result of this, the rejuvenating components the skin needs to maintain its appearance lessen. By replenishing lost peptides, cytokines & growth factors with the use of a topical product on the skin, we, through the day &night skin care set, are able to effectively re-engage the skins healing process.

The SC21 day & night stem cell skin care rejuvenation set also has a complete solution for restoring aging skin. We have, through the day anti-aging cream & night hydration serum been able to use: human mesenchymal stem cell growth factors, to regenerate human tissues; polypeptide complexes, (which penetrate the epidermis, outer layer of our skin) to send signals to the skin cells and cytokines proteins to send signals between the skin cells.

Focus Ingredient of Growth Factor Skin Care:

Mesenchymal Stem Cell (MSC) Peptide Complex = 15% (cytokines, growth factors, peptide complex)

Other Key Ingredients:

Focus Ingredient of Growth Factor Skin Care:

Mesenchymal Stem Cell (MSC) Peptide Complex = 20%(cytokines, growth factors, peptide complex)

Other Key Ingredients:

Apply 2-3 pumps to clean & dry skin.

Peptides are easier explained as signaling molecules produced by cells to instruct other cells.

As cellular messengers, cytokines influence and control our biological processes from start to finish. There are hundreds of unique cytokines in the human body. Cells talk with cytokines to repair injury, repel microbes, fight infections, and develop immunity.

Growth factors, are, on the other hand, diffusible signaling proteins that stimulate the growth of specific tissues and play a crucial role in promoting cell differentiation and division.

Many modern medical advances, including stem cell breakthroughs, are made possible due to our growing understanding of cytokines & growth factors. We use modern culture techniques (the same type used to produce human insulin and other naturally occurring substances) to grow human stem cells in the laboratory to harvest their regenerative cytokines and growth factors.

Mesenchymal stem cells (MSCs), which are traditionally found in the bone marrow, are used to improve function upon integration because they are self-renewing cells that have the capacity to differentiate, and are capable of replacing and repairing damaged tissues.

MSCs can consequently during culture, produce the following:

Our skin cells are biologically designed to continuously renew themselves, but, starting from our mid 20s, the skin cell renewal process slows down and our skin becomes thinner. This thinning causes us to be more prone to skin damage from external elements.

However, there are other factors that can contribute to our aging process, and in other cases even cause premature aging. Some of these factors include:

More:
Stem Cell Skin Care - anti-aging cream and hydration Serum

Recommendation and review posted by Bethany Smith

On 6 November 1998, the world woke to news of an astonishing discovery. James Thomson and his colleagues at the University of Wisconsin-Madison had generated stem cells from human embryos. Unlike other types of stem cells, these were pluripotent meaning they had the potential to generate any type of body tissue if given the right signals.

For many this news, and the accompanying claims that embryonic stem (ES) cells could revolutionise medicine, appeared to come out of the blue. However, for those of us already working in the stem cell space it was the vital next step in exploring the potential of stem cell science.

Back in 1998, I was a keen PhD student, part of the stem cell research effort at Monash University. I was trying to create pluripotent stem cells from the skin cells of a mouse. The idea was to first clone a mouse embryo from its skin cell and harvest the ES cells. In the lab next door, Ben Reubinoff had been working with Alan Trounson and Martin Pera for several years to see if they could make embryonic stem cells from donated human embryos effectively in parallel to their colleagues in Wisconsin.

There was a lot of excitement about how we might one day be able to use these cells to make replacement body tissues effectively on demand and alleviate suffering for many patients. Although we all recognised this was going to take an enormous amount of effort and time to deliver.

Outside the lab if I mentioned that I worked in stem cell research, I was met with overwhelming curiosity. But people also wondered why we couldnt just use adult stem cells which are found in some of our organs. Many people I spoke to already knew somebody who had been helped by a stem cell transplant using bone marrow or cord blood. Why did we need to use human embryos and ES cells at all?

The reason was, and still is, that adult stem cells are not able to generate any type of tissue because they are not pluripotent. Bone marrow stem cells, for instance, can regenerate an immune system but they cannot regenerate the pancreas or brain tissue. The only source of pluripotent cells was surplus human embryos originally created in an IVF clinic and then donated to research.

In 2007, Japanese scientists made a landmark discovery that side-stepped the need to use embryos. They were able to manipulate ordinary human skin cells to make them pluripotent (a much more elegant and effective approach than my attempts with mice skin cells during my PhD). Dubbed induced pluripotent stem cells or iPSC, these cells share the same desirable features as ES cells. They can be grown in the lab and coaxed to form specific types of body cells.

But both sources of pluripotent stem cells also carry the risk that they could form a tumour if we dont fully direct their developmental fate. Any clinical application must meticulously weed out the stem cells as part of the laboratory recipe used to make the replacement cells. For me, the crucial challenge is how to harness the potential of stem cells to develop safe and effective treatments.

These days, as the head of the outreach and policy program for Stem Cells Australia, a nationwide consortium of Australian stem cell scientists, I spend a lot of my time talking to the public. To some extent Ive become a race caller frequently asked to predict what new treatments are likely to come galloping down the track. Sometimes Im asked to offer an opinion on stem cell treatments that are not on the track at all. Promoted as a sure thing and available now for a price, these interventions lack credible evidence that they work or are even safe. Providers are effectively peddling hope and should be viewed with caution.

Fortunately, we do have providers committed to responsibly advancing the field with lots of bona fide contenders in clinical trials. So with my binoculars firmly in place, here is my reading of whats coming down the track.

Jeffrey Phillips

Leading the charge towards the clinic is a possible treatment for the most common cause of age-related vision loss: macular degeneration. In Australia about one in seven people over the age of 50 have some evidence of this disease. In this condition, damage to the cells at the back of the eye the macula affects central vision and the ability to read, drive and recognise faces. The actual seeing cells in the macula are intact but sight is lost because a tiny underlying patch of darkly pigmented cells are damaged. Known as retinal pigmented epithelial cells or RPE cells, they act like a pit stop team, feeding and clearing away waste for the highly active cells of the retina.

Because the number of RPE cells needed is very small and pluripotent stem cells readily develop into this exact tissue (you can easily spot a patch of darkly pigmented cells in the dish), macular degeneration has long been a favourite. Clinical trials are now underway in the United States, United Kingdom and Japan to determine whether replacing faulty RPE cells with those made in the lab from either human embryonic stem cells or induced pluripotent stem cells could help.

At this early stage, safety is a key concern. The surgical technique to deliver the cells carries the risk of detaching the retina and causing further vision loss. In May 2018, the London Project to Cure Blindness announced that two patients with macular degeneration specifically whats called the wet form due to extensive blood vessel growth under the retina had improved their vision with no significant side-effects after participating in a clinical trial.

Another early entrant in the race to the clinic is type 1 diabetes. Its a disease caused by friendly fire: the immune system seeks and destroys the beta cells of the pancreas. These remarkable cells can both sense rising blood sugar levels and release the exact amount of insulin needed to lower glucose levels to normal. When these cells are destroyed, which often occurs in childhood, the person is no longer able to control their blood sugar levels.

More than 120,000 Australians manage the disease with regular injections of insulin. But they cant regulate their blood sugar levels as precisely as beta cells do. And there are consequences: high blood sugar levels can damage the blood vessels in the heart, eyes and kidneys, while low levels can be fatal. Some patients have been lucky enough to receive a whole pancreas transplant or tissues containing beta cells from cadavers. But there are two problems. First, transplant donors are in short supply. Second, the donated tissue will likely suffer the fate of the original: attack by the immune system.

Enter pluripotent stem cells. Supply is no longer a problem. After two decades of trying, scientists are now able to make large quantities of fully functional beta cells in the lab. And as far as keeping the immune system at bay, several start-up companies have come up with the tea-bag approach. They encase the beta cells in a porous capsule. Like tea leaves, the beta cells are netted in but soluble factors easily move in and out across the net, including insulin and blood-borne glucose as well as other nutrients. Crucially, the net also stops marauding immune cells from getting to the beta cells.

The Californian company, Viacyte, is trialling a teabag about the size and shape of a credit card. Made of surgical-grade polymer, the capsule encases immature beta cells (theyre more robust if they mature inside the body), and is inserted just under the patients skin.

The key challenge, so far, is providing intimate contact with surrounding blood vessels so that the transplanted cells increase in number and survive. In June this year, the company reported its results at a meeting of the American Diabetes Association. Overall, they said there was a low rate of survival, but when cells did survive they produced insulin.

The company is now evaluating a second device that allows the patients blood vessels to grow through the walls of the capsule.

Jeffrey Phillips

A strong stayer in the race to the clinic is Parkinsons disease (PD). Predominantly a disease of ageing, around 1% of people over the age of 60 suffer from it.

The disease results from the death of brain neurons that release the neurotransmitter dopamine. Like a conductor, dopamine ensures different parts of the brain act in synchrony to execute routine movements. Without dopamine, patients have trouble controlling their walking and experience tremors in their hands and other parts of their bodies. Could replacing the faulty dopamine-producing neurons with healthy ones provide a way to combat PD?

More than 20 years ago, a few different research groups around the world gave it a try. Using human foetal tissue, they dissected out the dopamine-producing cells, and surgically implanted these into the brains of patients, specifically in a region called the striatum.

Some patients improved, but others reported significant side effects, particularly uncontrollable jerky movements known as dyskinesia. Questions were asked about whether the correct types of cells were being transferred to the correct part of the brain and further experiments were put on hold. A key question was whether pluripotent stem cells could offer a more precise and reliable source of dopamine-producing cells.

Jump forward to 2018 and several groups are on the cusp of testing new types of replacement cells for PD in a series of clinical trials. Years of research has shown that ES cells and iPS cells can be directed to develop into the correct type of neurons and that sufficiently large numbers can be generated.

When tested in animals, the dopamine-producing cells corrected movement disorders and did not form tumours.

This time around, rather than working in silos, different groups of researchers in Japan, Sweden, UK and US have banded together in a coalition called G-Force PD. Although each group is using a slightly different approach for their clinical trial, by sharing their results and expertise they hope to bring a cell-based therapy for PD closer to reality.

Jeffrey Phillips

Skin stem cells have long been solid performers for growing skin grafts to treat severe burns. But in November 2017, headlines ran hot with a report that a seven-year-old refugee Syrian boy, on the verge of death from a genetic skin condition, had been saved by a graft of skin stem cells corrected by gene therapy.

Hassan, now living with his family in Germany, suffered from a severe form of Epidermolysis Bullosa (EB). Its been referred to as the worst disease youve never heard of. It affects about 500,000 people worldwide, and can be caused by mutations to 18 different genes. In each case, the mutation disrupts the anchoring of the skins upper layer, the epidermis, to the underlying dermis. The result is skin that tears as easily as a butterflys wing. The only treatment is painful bandaging and re-bandaging.

Hassans skin had started blistering from birth but by the time he was seven, a bacterial infection had robbed him of 80% of his skin cover. In a last ditch effort to save his life, his German doctors contacted veteran stem cell researcher Michele De Luca at the University of Modena and Reggio Emilia in Italy. In 2006, De Luca had used skin grafts corrected by gene therapy to treat a leg wound of a woman who suffered from the same form of EB that Hassan suffered from. It was caused by a mutation to a gene called LAMB3.

De Lucas team took a tiny patch of skin containing stem cells from Hassans groin. They also spliced a copy of the LAMB3 gene into a benign virus. Then they infected the skin cells with the virus which ferried the LAMB3 gene into their DNA. The genetically corrected skin grew into a sheet which was grafted onto Hassans body. Five months after the first graft, Hassan was discharged. A month later he was back at school and playing soccer. Thanks to the genetically corrected stem cells, his grafted skin no longer blisters or shreds. The executive director of the Dystrophic Epidermolysis Bullosa Research Association of America dubbed Hassans treatment a sea change to the world of EB. Besides de Lucas group, Peter Marinkovich and Jean Tang at Stanford University School of Medicine, United States, are also trialling genetically-corrected skin grafts for a different type of EB.

Jeffrey Phillips

One of the front runners at the start of the stem cell race was spinal cord injury. Perhaps you remember the actor Christopher Reeve, aka Superman? Following a horse riding accident that left him a quadriplegic, he campaigned tirelessly for researchers to be allowed to use human embryonic stem cells to treat spinal cord injury which claims about 180,000 new cases each year. Perhaps thanks to his efforts in 2010, the world saw the first clinical trial using cells made from human ES cells.

Conducted by the California based biotech company Geron, the researchers had directed ES cells to develop into precursors of oligodendrocytes. These octopus-like cells wind their arms around neurons in the spinal cord to provide electrical insulation as well as nurturing factors. With a spinal cord injury, these important support cells can be lost. Four patients were injected with stem cell-derived oligodendrocyte precursors soon after their injury.

Controversially, Geron discontinued the study in 2011 to refocus their business. Asterias Biotherapeutics picked up the baton and last July, in a company press release, reported the results of an early clinical trial on 25 additional patients who were all injected with oligodendrocyte precursors three to six weeks post-injury. They reported no serious adverse events and that four patients recovered a degree of motor function that may increase their ability to lead an independent life. However, we have to wait to see the peer reviewed published results before we can assess the state of progress.

Beyond replacing oligodendrocytes made from ES cells, other clinical trials are testing different types of cells ranging from neurons obtained from donated foetal tissue to using the patients own cells obtained from the back of the nose where they play an important role in supporting the regeneration of the olfactory neurons. Some types of transplanted cells may act as paramedics, helping damaged motor neurons to recover. Others are designed to directly replace spinal cord neurons.

It remains too early to tell which approach will result in long-term improvements. While many with spinal cord injury are eager for even small improvements such as bladder or bowel control, patients should be careful about trying marketed experimental procedures outside well-conducted clinical trials as they may cause further harm. In a chilling example, one young woman who sought treatment using olfactory cells developed a large, painful mucus-secreting tumour in her spine and no improvement of her paraplegia. Unfortunately, many stem cell cures promoted online, especially for spinal cord injury, lack credibility.

Seeking advice from your medical specialist is the best way to find out more. If they dont know about a trial or claimed treatment, it is probably a mirage.

Jeffrey Phillips

Marked as a long shot for many years, stem cell research is starting to pay dividends for kidney disease. Though its not ready to provide transplants, it is already helping to discover new treatments.

Kidneys are the bodys vital cleansing and balancing system. They filter waste products and toxins from our blood into urine, maintain the bodys water balance and also make hormones important for regulating blood pressure and the production of red blood cells.

Kidney disease, which affects one in 10 Australians, damages the filtration units called nephrons. The major causes are diabetes and high blood pressure. Once gone, the nephrons cannot regenerate. But waiting for a donated kidney can take years; close to 1,000 Australians are currently on the waiting list for a transplant. This health crisis has catapulted researchers into trying to recreate kidney tissue from pluripotent stem cells an immense challenge as these are complex biological machines composed of many interacting parts.

Melissa Littles group, based at the Murdoch Childrens Research Institute in Melbourne, have pioneered this research. In 2015, they successfully grew tiny kidney-like structures that were showcased on the cover of Nature with the headline: Kidney in a dish. While their mini-kidneys possess many of the working parts of a mature kidney, theres a long way to go before they can be used as transplants. The plumbing for example bringing blood in and taking waste out is not yet functional. Also they are tiny, smaller than the tip of your finger.

Nevertheless, these mini-kidneys are already making a difference to our understanding of how kidneys develop and what goes awry in kidney disease, especially the hereditary form. For example, researchers were recently able to make mini-kidneys from a child suffering from a rare genetic condition that can cause end-stage kidney disease. They did it by first generating iPS cells from the childs skin. In the lab they were able to observe structural abnormalities in the childs cells and also showed that when the genetic mutation was corrected, the structural defect was corrected. This provides a new insight into inherited kidney disease where previously we knew very little about how these conditions develop.

Jeffrey Phillips

This article appeared in Cosmos 80 - Spring 2018 under the headline "The stem cell race"

More:
Which spare body parts will stem cells deliver first? | Cosmos

Recommendation and review posted by Bethany Smith

Alternative names: Gonadal Deficiency, Testosterone Deficiency

What is male hypogonadism? What are the signs of male hypogonadism? What causes male hypogonadism? How does my doctor tell if I have male hypogonadism? How is male hypogonadism treated?

Male hypogonadism is caused by a man's testes failing to produce normal levels of the male sex hormone, testosterone. Some men are born with hypogonadism, while others may develop the condition later in life.

There are two kinds of male hypogonadism:

Male hypogonadism at puberty can slow a boy's growth, and affect the development of normal male sexual characteristics. He may not undergo the normal changes a boy has during puberty, such as a deepening voice, body and facial hair, and increased muscle mass.

Male hypogonadism in adults can cause:

Primary hypogonadism, in which the testes do not work properly, can be caused by many conditions, including:

Secondary hypogonadism, in which the endocrine glands do not stimulate the testes to produce hormones, can be caused by:

Your doctor may check for low levels of testosterone (male sex hormone) by performing a blood test. He or she may also use blood tests to check the levels of the pituitary hormones (FSH and LH) that stimulate the testes to produce their hormones.

Other laboratory tests can help your doctor tell if hypogonadism is being caused by a problem with the testes, or with the pituitary gland. Such tests include:

If male hypogonadism is caused by a pituitary or other tumor, treatment is aimed at removing the tumor, or reducing its effects. This can include medication, surgery, and/or radiation therapy.

Male hormone replacement therapy has been used successfully for years to treat male hypogonadism. This involves a man taking testosterone by injection, transdermal system (patch), or gel.

Information on the Dartmouth-Hitchcockwebsite:

Our goals are to provide people with meaningful information to make informed decisions about their health and health care.

Dartmouth-Hitchcock and its affiliated component organizations aspire to deliver consistent high quality medical care to all patients and to continually improve its quality of care as evolving technology and medical knowledge permits.

Please call 911 in the case of any medical emergency.

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Male Hypogonadism | Endocrinology | Dartmouth-Hitchcock

Recommendation and review posted by Bethany Smith

In this video Paul Andersen explains how the CRISPR/Cas immune system was identified in bacteria and how the CRISPR/Cas9 system was developed to edit genomes.

Do you speak another language? Help me translate my videos:http://www.bozemanscience.com/transla...

Music Attribution

Intro Title: I4dsong_loop_main.wavArtist: CosmicDLink to sound: http://www.freesound.org/people/Cosmi...Creative Commons Atribution License

OutroTitle: String TheoryArtist: Herman Jollyhttp://sunsetvalley.bandcamp.com/trac...

All of the images are licensed under creative commons and public domain licensing:Adenosine. (2009). English: Artistic rendering of a T4 bacteriophage. The colours grey and orange do not signify anything, they are just used to illustrate structure. Created for Wikipedia. Retrieved from https://commons.wikimedia.org/wiki/Fi...E. coli Bacteria. (n.d.). Retrieved February 17, 2016, from https://www.flickr.com/photos/niaid/1...Fioretti, B. F. Hallbauer &. (2015). English: Director, Max Planck Institute for Infection Biology, Department of Regulation in Infection Biology. Visiting professor The Laboratory for Molecular Infection Medicine Sweden MIMS; http://www.mpiib-berlin.mpg.de/resear.... Retrieved from https://commons.wikimedia.org/wiki/Fi...Foresman, P. S. ([object HTMLTableCellElement]). English: Line art drawing of a chimera. Retrieved from https://commons.wikimedia.org/wiki/Fi...Magladem96. (2014). English: Picture of DNA Base Flipping. Retrieved from https://commons.wikimedia.org/wiki/Fi...project, C. wiki. (2014). English: Crystal Structure of Cas9 bound to DNA based on the Anders et al 2014 Nature paper. Rendition was performed using UCSFs chimera software. Retrieved from https://commons.wikimedia.org/wiki/Fi...Providers, P. C. (1979). English: Photomicrograph of Streptococcus pyogenes bacteria, 900x Mag. A pus specimen, viewed using Pappenheims stain. Last century, infections by S. pyogenes claimed many lives especially since the organism was the most important cause of puerperal fever and scarlet fever. Streptococci. Retrieved from https://commons.wikimedia.org/wiki/Fi...RRZEicons. (2010). English: zipper, open, close. Retrieved from https://commons.wikimedia.org/wiki/Fi...UC Berkeley. (n.d.). Gene editing with CRISPR-Cas9. Retrieved from https://www.youtube.com/watch?v=avM1Y...

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What is CRISPR?

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Major funding for "Making Stuff" is provided by the National Science Foundation.

This material is based upon work supported by the National Science Foundation under Grant No. DRL-1222986. Any opinions, findings, and conclusions or recommendations expressed in this material are those of the author(s) and do not necessarily reflect the views of the National Science Foundation.

Additional funding is provided by the U.S. Department of Energy's Office of Science.

This material is based upon work supported by the Department of Energy under Award Number(s) DE-SC0008715. This report was prepared as an account of work sponsored by an agency of the United States Government. Neither the United States Government nor any agency thereof, nor any of their employees, makes any warranty, express or implied, or assumes any legal liability or responsibility for the accuracy, completeness, or usefulness of any information, apparatus, product, or process disclosed, or represents that its use would not infringe privately owned rights. Reference herein to any specific commercial product, process, or service by trade name, trademark, manufacturer, or otherwise does not necessarily constitute or imply its endorsement, recommendation, or favoring by the United States Government or any agency thereof. The views and opinions of authors expressed herein do not necessarily state or reflect those of the United States Government or any agency thereof.

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NOVA - Official Website | Cryonics

Recommendation and review posted by Bethany Smith

Cryonics is the low-level temperature preservation of humans and animals in a suspended animation by slowing their vital functions, for the purposes of preserving them and keeping them alive for periods of time ranging from decades or even centuries until they are needed. Other purposes include safekeeping, or for keeping them alive for as long as possible while their brains are connected to an external expression device.

Before the Great War, Robert House and his company, as well as the Big Mountain Research Facility, are known to have produced technology within the cryogenic and cryonic field. Robert House extensively researched extending the human life, and created his own cryonic chamber that allowed the user to connect their brain pattern and consciousness to an external interface. Big Mountain produced hibernation chambers with similar purposes, however, the nature of their research into the field is unknown.[1] The United States Armed Forces experimented with cryonic technology as well, making the systems at the Raven Rock facility, and using cryonic chambers in the Sierra Army Depot.

The Sierra Army Depot dealt in the field of cryonics, with one example being their cryonic submergence of Dobbs in bio med gel.

The Environmental Protection Agency made use of hibernation chambers, holding three subjects within each. These editions were large transparent tanks filled with ice cold water, that is filled and drained through a long serpentine pipe system.

The Enclave's scientific personnel at the Raven Rock base in the Capital Wasteland have several rooms dedicated to stasis, and their prisoner restraints use the same systems. There are two different versions of the Raven Rock stasis chambers: a green chamber holding the subject in the center, and a transparent blue holographic/light-based/photonic resonance chamber. The systems in Raven Rock appear to be the most advanced systems of their kind currently known on Earth. These chambers have been used to preserve yao guai, deathclaws, super mutants, feral ghouls, and human prisoners. Whether these stasis chambers are built for long-term preservation on the scale of years is unknown. The same blue-light stasis technology is used in Fort Constantine's T-51b power armor storage room. Although unseen in-game, Vault 87 was meant to have been equipped with four stasis-chambers.[2]

Its history unknown, a liquid nitrogen-based weapon commonly known as a Cryolator could be easily constructed.

The alien civilization aboard Mothership Zeta made cryonics a core aspect of their invasion and their research. The longest known preservation of a subject aboard the ship was over 600 years. They worked by entirely sealing the subject and the chamber from the outside, and used freezing cold air to suspend the subject. When opening, the chamber would decompress and stabilize with outside air, and force the subject to drop to the floor. Furthermore, there was a second method of cryonics used by the aliens. In their dedicated cryo lab, during the Zeta Uprising, the Lone Wanderer could have frozen alien soldiers with the loose systems, thawing them into an ice block for a mere few seconds. Elliott Tercorien could have harnessed the energy to create cryo grenades and cryo mines, which exact the same effect on targets within the blast radius.

The only instance of cryonic technology in the Mojave Wasteland is Robert House's cryonic preservation chamber in the Lucky 38. This is one of the only known instances of a cryonic chamber that links the subject's brain and consciousness with an external interface. It is an advanced piece of technology, linking Robert House to an external interface, from which he has access to large amounts of data from the Lucky 38's mainframe, as well as the ability to control his Securitrons. Opening the chamber, even for a second, will doom the subject to having only little more than a year left at life due to exposure to outside contaminants. House hopes that with the Courier's help, he will be able to make the same cryonic technology he uses available to other high-value individuals in the future. The only other instance of cryonic technology is at the hazmat testing ground in the Big Empty, for storing the hazmat suit

Vault 111, located in Boston, was built to observe the effects of suspended animation on unsuspecting test subjects for 180 days, and holds multiple cryosleep pods. They are first shown to the Sole Survivor pre-War, disguised as "decontamination" pods. The Sole Survivor was preserved for exactly 210 years, with their spouse and son being taken in the year 2227, 150 years in. All the other residents of the Vault perished by 2227, due to Conrad Kellogg not reactivating their life support, with Shaun being abducted as an infant by him for the Institute.

Green stasis tanks are also seen holding super mutants in the FEV Lab in the Institute's Bioscience sector.

Vault 0 kept pre-War geniuses in cryogenic stasis, by extracting their brains from their body and freezing them. They were then hooked up to the Calculator supercomputer, melding all of the identities of each connected brain into one.

The Boulder Dome in Denver was equipped with prototype military medical cryo tanks that had a very high malfunction rate, and before the Great War, scientists were frozen in sleeper tanks. Victor Presper continued to use the equipment during his time spent there.

Cryonic technology appears in Fallout 2, Fallout 3, its add-on Mothership Zeta, Fallout: New Vegas and its add-on Old World Blues, Fallout 4, and Fallout Tactics. It is also mentioned in the Fallout: New Vegas add-on Dead Money. Cryonic technology was also meant to appear in the canceled Van Buren.

Originally posted here:
Cryonics | Fallout Wiki | FANDOM powered by Wikia

Recommendation and review posted by Bethany Smith

Several genes are known to be associated with higher risk of Alzheimers disease (conversely, in some cases these genes have been found to protect against the disease). One of these genes is known as APOE.

APOE has three common forms:

APOE2, the least common gene, is believed to play a role in reducing the risk of Alzheimers diseaseAPOE4, more common than APOE2, is believed to increase the risk of Alzheimers diseaseAPOE3, the most common gene, has not been found to increase or decrease the risk of developing Alzheimers disease

Inheriting one of the APOE4 gene variants found in about 20% of the populationmay increase the chance of developing Alzheimers disease by a factor of four. Inheriting two of APOE4 variants (one from each parent) will increase the chance by a factor of 10. Additionally, a 2014 study found that women with the APOE4 gene were twice as likely to get Alzheimers disease than women who do not carry the gene; for men with the APOE4 gene the risk factor does not increase.

Known genetic factors such as this account for a small percentage of all Alzheimers disease cases, but current research supported by Cure Alzheimers Fund and others indicates that genetics have a far greater influence than was previously thought. Additional candidate genes are being discovered and studied to determine their possible role in the disease. The genes we do know about account for a large percentage of early-onset cases: The rare Presenilin 1 and Presenilin 2 genes, for example, virtually guarantee development of Early Onset Alzheimers disease.

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Female genetics - Women and Alzheimer's

Recommendation and review posted by Bethany Smith

Cryonics is a fascinating concept that has inspired the imagination and dreams of thousands of people worldwide. If you're someone interested in the theory, remarkable potential and practical applications of cryonics, joining CI is a great way to learn more and get involved in the cryonics movement. Our members come from all walks of life and from all around the world, united by our interest in cryonics and the potential benefits it holds for ourselves and for all mankind.

As a member, you will be one of the owners and operators of the Cryonics Institute, as we are wholly owned and operated by our membership. CI is run by a Board of Directors elected exclusively from our members, by our members - so we have no outside investors, managers or other parties dictating our operations. Our responsibilities are to our patients, our membership, and to advancing our founder Robert Ettinger's vision. CI membership is an excellent starting point for anyone interested in cryonics to learn more and be part of an exciting, potentially world-changing community of forward-thinking people. There are no specific duties or formal responsibilities required for membership, apart from applying and paying the membership fees. However a large number of our members take a more active role in the organization either as officially elected Officers or as volunteers. How active you choose to be is completely up to your own discretion.

Please note, Cryonics Institute Membership is required if you are actively planning cryonic suspension services for yourself or a loved one through CI. Our "Members-Only" policy for cryonic services helps ensure the quality of our suspensions, and maintains the integrity of our organization and operations. CI is our organization and as member-owners it's clearly in our own best interests to manage it efficiently and especially to insure the highest standards for our suspension arrangements.

There are two classes of CI Membership. A Lifetime Member pays a one-time fee of $1,250 and can arrange for cryopreservation at CI for $28,000, usually by making CI the beneficiary of a life insurance policy. Other close family members can join for an additional $625 (there is no charge for minor children). An Annual (or Yearly) Member pays a $75 initiation fee plus $120 yearly (or $35 quarterly) and can arrange for cryopreservation at CI for $35,000. Every Yearly Membership family member must pay the same price. Neither of these fees include the cost of preparation or shipment by a local funeral director, which must be arranged separately (often with a Local Help Rider). To join, simply fill out a membership form for the type of membership you desire, Annual ($120/year recurring) or Lifetime ($1,250 one time.) The forms are available below or can be mailed on request.

To learn more about membership options and details, please see our Frequently Asked Questions. We also provide a special Membership Outreach program that gives you the opportunity to speak one-on-one with a current CI member who will help answer your questions via phone or email.

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Membership | Cryonics Institute

Recommendation and review posted by Bethany Smith

NOTE: Forever Health provides a directory where people like you can connect with BHRT and other physicians and innovative health practitioners. We do not provide medical advice or services directly. While BHRT and prevention are important to the physicians and health practitioners listed, each has his or her own approach to practicing medicine. So when scheduling your appointment, be sure to clarify the reason for your visit, as well as your goals for seeking out such treatment.

NOTE on Insurance: Innovative practitioners and insurance companies have long debated the importance of preventive medicine and services such as BHRT. Due to variations in coverage for novel treatment options, many practitioners will resolve this issue by providing their services on a cash-only basis. Forever Health encourages you to contact the practitioner to determine if any alternative payment options or post-visit reimbursements exist.

DISCLAIMER: Inclusion in this directory is free to practitioners and does not constitute endorsement by Forever Health. All health practitioners who appear on this list do so on the sole basis of their own expression of interest in the fields of BHRT or other integrative medicine. Forever Health does not verify the competence, professional credentials, business practices or validity of the expressed interests of these health practitioners. Forever Health makes no recommendation of any health practitioner on this list and makes no suggestion that any such health practitioner will cure, treat, or prevent any disease or condition.

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Bioidentical Hormone Doctors

Recommendation and review posted by Bethany Smith

iPSCells Represent a Superior Approach

iPS cell-derived cardiomyocyte patch demonstrates spontaneous and synchronized contractions after 4 days in culture.

One of the greatest promises of human stem cells is to transform these early-stage cells into treatments for devastating diseases. Stem cells can potentially be used to repair damaged human tissues and to bioengineer transplantable human organs using various technologies, such as 3D printing. Using stem cells derived from another person (allogeneic transplantation) or from the patient (autologous transplantation), research efforts are underway to develop new therapies for historically difficult to treat conditions. In the past, adult stem and progenitor cells were used, but the differentiation of these cell types has proven to be difficult to control. Initial clinical trials using induced pluripotent stem (iPS) cells indicate that they are far superior for cellular therapy applications because they are better suited to scientific manipulation.

CDIs iPS cell-derived iCell and MyCell products are integral to the development of a range ofcell therapyapplications. A study using iCell Cardiomyocytesas part of a cardiac patch designed to treat heart failure is now underway. This tissue-engineered implantable patch mayemerge as apotential myocardial regeneration treatment.

Another study done with iPS cell-derived cells and kidney structures has marked an important first step towards regenerating, and eventually transplanting, a functioning human organ. In this work, iCell Endothelial Cellswere used to help to recapitulatethe blood supply of a laboratory-generated kidney scaffold. This type of outcome will be crucial for circulation and nutrient distribution in any rebuilt organ.

iCell Endothelial Cells revascularize kidney tissue. (Data courtesy of Dr. Jason Wertheim, Northwestern University)

CDI and its partners are leveraging iPS cell-derived human retinal pigment epithelial (RPE) cells to develop and manufacture autologous treatments for dry age-related macular degeneration (AMD). The mature RPE cells will be derivedfrom the patients own blood cells using CDIs MyCell process. Ifapproved by the FDA, this autologous cellular therapy wouldbe one of the first of its kind in the U.S.

Learn more about the technologybehind the development of these iPScell-derived cellular therapies.

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Cellular Therapy - fujifilmcdi.com

Recommendation and review posted by Bethany Smith

Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patients cells instead of using drugs or surgery. Researchers are testing several approaches to gene therapy, including:

Replacing a mutated gene that causes disease with a healthy copy of the gene.

Inactivating, or knocking out, a mutated gene that is functioning improperly.

Introducing a new gene into the body to help fight a disease.

Although gene therapy is a promising treatment option for a number of diseases (including inherited disorders, some types of cancer, and certain viral infections), the technique remains risky and is still under study to make sure that it will be safe and effective. Gene therapy is currently being tested only for diseases that have no other cures.

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What is gene therapy? - Genetics Home Reference - NIH

Recommendation and review posted by Bethany Smith

One of the bravest moves in that direction has come from stem cell research and therapy. Stem cell therapy is currently being used to successfully treat more than 80 diseases, but the field is rapidly evolving backed by prestigious research and clinical trials.

Smart Cells is the first private UK stem cell storage company to have released stored stem cell units for use in the treatment of children with life-threatening illnesses. We have released the greatest number of samples for use in transplants from the UK.

We believe with the development of technology in the future we will be able to treat even more illnesses.

We believe our customers deserve the best service available and we run our state of the art facility with leading professionals in the field.

We believe that storing your childs stem cells at birth can be a crucial part of treating or curing an unexpected illness.

We believe that in the future this service should be available to every parent, child and family.

We are a company that is for life.

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Storing Stem Cells For Life - Smart Cells

Recommendation and review posted by sam

Youve been using Natural Hormones all your life, why change now?

The term Bio-identical Hormone Replacement Therapy is interchangable with Natural Hormone Replacement Therapy.

Health is a holistic goal, and hormonal health put simply, is a large significant factor of that big picture. To control your hormonal health you need towork with an understanding doctorwho believes in the value of hormonal testing to use as a tool in the treatment of your health.

Combining your symptoms with your hormonal profile, looking at you individually, and considering your own, and your family history a picture can be brought into focus. Once obtained, this picture of your individuality can be used to give you a predictable and active future so that age is not a barrier to wellness.

When hormone supplementation is warranted, there arefour major points to consider. These stem from the belief that if we are going to relieve or reverse the ageing process, it should be done in such a way as to mimic the bodys system as it was created. In that endeavour, only hormones that are the exact molecules made by the human endocrine system should be used. The human race would not have survived this long if these hormones were dangerous and subjected us to fatal diseases.

Secondly, those hormones used should be introduced into the bloodstream in a way that emulates the glands as closely as possible, that is, avoiding the digestive tract and liver and minimising first-pass effects.

Thirdlya broader spectrum of hormones, at lower doses, offers a more complete, physiological balance.

Finallythe dose must be individualised to the patients needs and goals which in the end will result in fewer side effects if any, along with reducing or eliminating other medical problems.

Bio-identical Hormone Replacement Therapyproducts are compounded by a compounding pharmacist, which offers you and your physician a choice for individualised hormone replacement.

Naturalrefers to thestructureof the hormone (i.e. they are bio-identical in structure and function to the hormones naturally produced in our bodies). The hormones start from wild yam and soybeans which are rich in precursor molecules. These are easily converted by biochemists into other molecules that areidentical in all aspectsto our own naturally occurring hormones.

It should be emphasised that although these precursor molecules can be synthesised into natural bio-identical hormones in a test tube, our bodies are incapable of doing so. Therefore although wild yam creams are good moisturisers they do nothing to increase our hormonal levels.

Insulin used by diabetics was obtained in the past from pigs, which saved many lives, yet caused a significant amount of side effects and problems. Pig insulin is no longer used by diabetics as a Natural identical human insulin has been established, overriding any reason for using pig insulin. In this same light, there is now NO cause for women to be using oestrogens and progesterones produced from the urine of pregnant mares, aswe now have natural bio-identical human hormones available.

Bio-identical hormone replacement therapy is administered generally, in the form of atroche(lozenge) or as a transdermal cream which areabsorbed into the blood streamwithout having to go through the digestive system creating stress on the liver. We find Troches are the most suited form for Natural Hormone Replacement Therapy mainly because of the ease in which the ingredients in each prescription can be altered, and because of their superior transbuccal absorption.

Compare this to the mass produced formulas available commercially where titrating a dose is almost impossible and one must fit in with what is available.

Results have been good to excellent and in many cases extraordinary. The natural or bio-identical hormones used in the treatment of menopause are any combination of natural Progesterone, Oestrogen, Testosterone, at levels determined specifically for each patient.

Trochescan be mixed so that each individual troche contains a combination of various natural bio-identical hormones in small doses. For example, a troche can be made containing a mixture of any of the hormones, eg: oestrogen, progesterone, testosterone in any possible doses according to your needs. This combination is used to treat the symptoms of menopause.

Small doses oftestosteroneare useful inmenopausefor depression, to enhance energy levels, mood and libido without causing side effects.

Troches containing natural progesterone alone are helpful for premenstrual syndrome and peri menopause and can be taken during the latter half of the menstrual cycle to alleviate depression, migraine, nausea and basically all those PMT symptoms that occur premenstrually.

Menopausal women who have had ahysterectomycan benefit from troches containingnatural bio-identical estrogens,progesteroneandtestosterone. Even though the uterus may not be present, in the case of hysterectomy,progesterone is still essentialto balance the oestrogen component and stop oestrogenic side effects. Your requirements should be determined with a blood test to measure your sex hormones we recommend tests for blood oestrogen, progesterone and testosterone.

Troches can be made palatable, by making them in different flavours. Each troche is made up on an individual basis and the whole process takes a few days.

Once deciding to embark on taking hormone replacement one is usually forced to grapple with the risk/benefits factor. It must be stressed that although we believe Bio-identical hormones used in troches may be safer than Synthetic hormones,there are still risksrelating to the use of natural hormones. These risks are mostly related to the uses of oestrogen and the increase risk of breast cancer. For medicolegal purposes we have to assume the use of bio-identical hormones carries the same risk as synthetic hormones. The current thinking suggests that if we need to use any form of oestrogen to treat menopause we should attempt to stop this within 2 yrs of commencement.

Tailor made troches allow slow reduction of oestrogen and in my experience it is most definitely easier to slowly wean a patient off Oestrogen due to ease of dose adjustment. It would appear that continuation on progesterone and testosterone over a longer period is a safer option.

The rest is here:
Bio-Identical Hormone Replacement Therapy - Hormone Clinic

Recommendation and review posted by Bethany Smith