captionLuxury Travel Magazine predicts that ancestry travel will be one of 2020s hottest travel trends.sourceXurxo Lobato/Getty Images

Over the past decade, millions of people have used genetics kits to identify their roots.

Since launching in 2007 and 2012, industry leaders 23andMe and AncestryDNA have gained 10 million and 15 million users respectively.

The process for consumers is a simple one: Spit into the kit tube, send it off in the mail, and within a few weeks, receive a detailed report on where your ancestors came from or in the case of 23andMe, genetic predispositions as well. The kits are also affordable: AncestryDNAs currently start at $59, and 23andMes start at $79.

The golden age of at-home DNA tests, however, may be nearing its end. Earlier this week, Business Insiders Lydia Ramsey reported that the demand for these sub-$100 tests is plateauing.

The fads over, Luke Sergott, an analyst at Evercore ISI, told Ramsey. Sergott noted that many people have taken the test and are looking for the next best thing. Concerns over privacy and ethics are two other reasons why sales may be slowing.

One trend that these DNA tests have spawned does not seem to be seeing the same slowdown: Ancestry tourism or traveling to the places where your family and ancestors lived has been on the rise, and its still gaining momentum.

Ancestry travel has experienced a boom over the past several years.

In fall 2017, Go Ahead Tours and Ancestry DNA partnered to offer customized heritage trips featuring hand-picked hotels and on-hand Ancestry genealogists. In 2018, luxury cruise ship operator Cunard launched its Journey of Genealogy series in collaboration AncestryDNA. The inaugural trip was a seven-night cruise from Southampton in the United Kingdom to New Yorks Ellis Island.

And in May, Airbnb announced a partnership with 23andMe in which the hospitality giant provides users with custom travel and experience recommendations.

DNA travel has become increasingly popular, Airbnb said in its announcement of the collaboration: Since 2014, the number of travelers using Airbnb for tracing their roots increased by 500 percent, and 78 percent of these trips are taken in pairs or solo, suggesting that these are introspective journeys or an important moment to share with a significant other.

Independent tour operators have also hopped on the ancestry travel bandwagon with new heritage-based tour offerings. Small-group adventure travel company Classic Journeys offers to match travelers with trips after reviewing their DNA results from23andMe, AncestryDNA or another provider. Luxury service The Conte Club designs travel experiences that can span weeks and cost as much as $132,000 based on DNA tests.

According to Luxury Travel Magazines travel predictions for the new decade, ancestry travel is one of 2020s fastest-growing sectors.

In May, Airbnb noted that travelers who have expressed the most interest in ancestry tourism are from places typically known for their history of immigration. These include the U.S., Canada, Australia, mainland China, the UK, France, Korea, New Zealand, Taiwan, and Brazil.

Ahead of the 75th anniversary of the end of World War II, a time when many Poles fled Eastern Europe, Poland is preparing for an uptick in ancestry travelers. Over a dozen new hotels, including a new branch of Robert De Niros Nobu Hotels, are set to open in Warsaw next year, Luxury Travel Magazine reports.

Cunard is looking ahead to another immigration anniversary: the quatercentenary of the Mayflower voyage. In August, the cruise ship operator will be offering a journey on the Queen Mary 2 that replicates the English Puritans journey to the New World.

While Airbnb says travelers between the ages of 60 and 90 are most likely to take heritage travel trips, the preference for ancestry travel may be skewing younger.

[Home DNA tests are] much more approachable to the younger generation than sitting in front of a computer screen or [microfilm reader] trying to do family history, Dallen Timothy, a professor at Arizona State University and editor of the Journal of Heritage Tourism told Business Destinations in April. This trend is likely broadening the genealogy tourism market by attracting younger generations.

Travelers are transitioning from looking for local experiences to seeking experiences that fundamentally change them, Chris Roche, business director of luxury safari company Wilderness Holdings, told Business Insiders Katie Warren.

Experiential wouldve been the buzzword five years ago, Roche said. The last couple of years, thats transitioned into transformational.'

This search for meaning is redefining the travel space, Warren noted, citing the Global Wellness Summits 2018 trends report.

Robin Hauck, director of Business Development and Partnerships with Go Ahead Tours, told NBC last winter that AncestryDNA had been hearing from an increasing number of customers that they wanted to bring the results of their kit to life. Its just so important for people to fill in in actual living color where theyre from and how their ancestors lived. It makes people feel more complete, he said.

Rebecca Fielding, CEO of The Conte Club, echoed Hauck in conversation with Conde Nast Traveller, noting that ancestry trips are a deep dive into ones identity.

It can be a complicated and emotional process, but the journey into our own past might be the most meaningful trip we can take, she said.

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The golden age of at-home genetic testing may be over but its still a critical part of one of the biggest trends in how people will be traveling in...

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If youre going through IVF, you may be offered a test to look at your embryos chromosomes.

Pre-implantation genetic testing for aneuploidy (chromosome abnormalities), known as PGT-A, is an add on used to help choose embryos with the right number of chromosomes. Its promoted by IVF clinics as a way to increase the chance of success, especially for women over 35.

But the evidence shows that in most cases, PGT-A doesnt improve the chance of a baby.

Read more: The business of IVF: how human eggs went from simple cells to a valuable commodity

Human cells usually contain 46 chromosomes. Aneuploidy is a term that describes a chromosome number that is different from 46 either too many or too few chromosomes.

In human embryos, most aneuploidies are lethal, resulting in miscarriage, or do not result in pregnancy at all.

The chance of aneuploidy increases with the age of the woman; by the time a woman reaches age 40, approximately 80% of her embryos are aneuploid.

All couples produce some aneuploid embryos, whether they conceive naturally or with IVF. The idea behind PGT-A is that if the aneuploid embryos can be identified they can be discarded, so that only embryos capable of producing a healthy pregnancy are used.

PGT-A involves the woman having fertility drugs to produce several eggs. When they are mature, they are retrieved and mixed with sperm to create embryos.

Embryos are grown in the laboratory for five to six days. At this time, two types of cells are distinguishable: the cells that will develop into the placenta and the cells that will become the baby.

Read more: Considering using IVF to have a baby? Here's what you need to know

A few cells are removed from the future placenta for testing and the embryos are frozen until test results are available.

If the test shows there are normal embryos, one is thawed and transferred to the womans uterus. Any remaining normal embryos will be kept frozen for transfer later if the first transfer is unsuccessful.

Importantly, PGT-A doesnt correct chromosomally abnormal embryos, it simply allows couples to avoid transferring them.

Many clinics recommend PGT-A for women over 35 (more than half of women who have IVF) and those who have had repeated miscarriages or failed IVF treatments. This is because women over 35 and women with previous losses are more likely to produce aneuploid embryos.

While the theory behind PFT-A makes sense, randomised controlled trials (the gold standard evidence to tell us if an intervention makes a difference) have not demonstrated a clear benefit.

Of the two most recent trials of PGT-A, one reported fewer embryo transfers and fewer miscarriages in the PGT-A group but neither showed benefits in terms of improving the live-birth rate.

PGT-A actually has the potential to reduce the chance of a baby. It can do this in two ways.

First, PGT-A is not 100% accurate. This means that inevitably, some embryos that have the capacity to form a healthy baby will be discarded.

The most common reason for these false positive results is that a proportion of embryos are mosaic they have a mix of normal and abnormal cells. Surprisingly, mosaic chromosome abnormalities are quite common in early human embryos, and do not seem to prevent the embryo developing into a healthy baby.

However, if abnormal cells are removed and tested, the embryo will be misclassified as abnormal and discarded a lost opportunity for a healthy pregnancy.

Read more: Fertility miracle or fake news? Understanding which IVF 'add-ons' really work

Many healthy babies have been born to people who have elected to have mosaic embryos transferred because they were the only embryos they had.

In a recent study of 98 women who had mosaic embryos, 32 (33%) elected to have at least one transferred. Of these, 11 (34%) had a successful pregnancy with apparently healthy babies born.

Second, while the risk is small, embryos can be damaged in the biopsy procedure and some embryos dont survive the freezing and thawing process.

PGT-A costs around A$700 per embryo which adds up to A$2,800 if there are four embryos to test.

While doctors likely offer their patients detailed and individualised information about different treatment options, information about the possible benefits of PGT-A on clinic websites can be difficult to interpret.

Thats why independent information about the pros and cons of PGT-A is needed to help people make informed decisions. The Victorian Assisted Reproductive Treatment Authority (VARTA) has developed a downloadable resource about the current state of knowledge about PGT-A.

Some clinics are now offering a less invasive technique where, rather than removing cells from the embryo, they test the fluid that the embryo is grown in to determine if the embryo has the right number of chromosomes. Time will tell of this will improve the chance of having a baby with IVF.

In the meantime, it may help to ask the five questions recommended by Choosing Wisely:

And in the case of IVF: how will this improve my chance of a live birth?

Read more: Your questions answered on donor conception and IVF

See original here:
Genetic testing IVF embryos doesn't improve the chance of a baby - The Conversation AU

Recommendation and review posted by Bethany Smith

A mother and daughter shared a special Thanksgiving Thursday in western North Carolina. The two are crediting genetic testing for uniting them.Kristy Wilson was put up for adoption at birth. Her mother, Sandra Derr, didn't think she'd see her again."No, never," Derr said. But several decades years later they used 23 and Me, a genetic testing service. The two say a hit came back, signaling a possible mother-daughter match. "It was pretty scary, because you don't know what to expect," Wilson said.The two communicated after the match and met in April. Wilson lives in Black Mountain, North Carolina while Derr lives in Delaware. "It was unreal, really, I just knew walking up this is the gal," Derr said. Wilson and Derr celebrated Thanksgiving at Wilsons house Thursday with 15-20 other people. "Immerse herself in my craziness for Thanksgiving, Wilson said."I'm so proud of who she is," Derr said. The two are united with a future ahead."I think it looks great, said Derr. Absolutely great."

A mother and daughter shared a special Thanksgiving Thursday in western North Carolina.

The two are crediting genetic testing for uniting them.

Kristy Wilson was put up for adoption at birth. Her mother, Sandra Derr, didn't think she'd see her again.

"No, never," Derr said.

But several decades years later they used 23 and Me, a genetic testing service. The two say a hit came back, signaling a possible mother-daughter match.

"It was pretty scary, because you don't know what to expect," Wilson said.

The two communicated after the match and met in April. Wilson lives in Black Mountain, North Carolina while Derr lives in Delaware.

"It was unreal, really, I just knew walking up this is the gal," Derr said.

Wilson and Derr celebrated Thanksgiving at Wilsons house Thursday with 15-20 other people.

"Immerse herself in my craziness for Thanksgiving, Wilson said.

"I'm so proud of who she is," Derr said.

The two are united with a future ahead.

"I think it looks great, said Derr. Absolutely great."

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52-year-old daughter celebrates first Thanksgiving with mother - WYFF4 Greenville

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DENVER Airports are filled with family reunions during the holidays, but for the Robertson's, they could have never seen this one coming.

Michael Robertson said that back in August, his dad told him he had news.

"My dad called me one night and said, 'I have an email for you to read.'"

It was from a man named Robb Collins who found Robertson after using the DNA genetic testing and analysis service, 23andMe.

Eventually Robertson and Collins got in contact and found out they were long lost brothers.

Robertson's father served in the Navy and when visiting the Manila, met Collins' mother. After leaving Manila, Collins was born but said his mother couldn't get in touch with the father anymore.

"She explained that it was just... she lost track of him because he was in the Navy and its the 80s, so there was no Facebook," Robertson said.

For the first time and on Thanksgiving, Collins got to meet his father, brother and sister in person. A reunion he had only dreamed about for years and never thought would happen.

"I sort of look similar to my mom and whatnot and to see old pictures of him and to see pictures of my brother and to realize that Ive kind of been looking in the mirror my whole life is just mind blowing."

Continued here:
Man meets lost family on Thanksgiving thanks to 23andMe - The Denver Channel

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Spitting intotheplastic test tube, I felt nervous. I was offering up a piece of myself for decoding, and while this timethere was no silver-haired sage, it reminded me of a visit to a fortune teller when I was 21.

Then, I offeredthepalm of my hand in a bid to divine what fate had planned for me. Now, it wasDNA, with my saliva destined for a laboratory in southwest China, totheheadquarters ofChengdu 23Mofang Biotechnology Co., a startup thats seeking to tap a boom in consumer genetics intheworlds most populous nation.

Rising awareness of genetically-linked diseases like Alzheimers and a natural human curiosity for insight intothefuture is fueling a global market for direct-to-consumerDNAtesting thats predicted totripleoverthenext six years. In China, wherethegovernment has embraced genetics as part of its push to become a scientific superpower,theindustry is expected to see US$405 million in sales by 2022, according to Beijing research firm EO Intelligence, an eight-fold increase from 2018. Some 4 million people will send away test tubes of spit in China this year, and I had just become one ofthem.

Not only was I entering a world where lack of regulation has spawned an entire industry devoted to identifyingthefuture talents of newborn babiesthroughtheir genes, I was handing over my genetic code to a country wherethegovernment has been accused of usingDNAtesting to profile minority groups a concern that hit home whentheresults showed I was a member of one.

I wanted to see whethertheburgeoning industry delivered on its claims in China, where scientists have gained international attention and criticism for pushingtheboundaries of genetics. And as a child of Vietnamese immigrants totheUS, Ive long been curious about my ancestry and genetic makeup.

To get an idea of how this phenomenon is playing out intheworlds two biggest consumer markets, I comparedtheDNAtesting experience of 23Mofang withthefirm CEO Zhou Kun says it was inspired by:23andMe Inc., one ofthebest known consumer genetics outfits intheUS.

PushingtheEnvelope

Thedifferences betweenthetwo companies are stark.

23andMe was co-founded byAnne Wojcicki, a Wall Street biotech analyst once married toGoogleco-founderSergey Brin.TheMountain View, California-based firm has more than 10 million customers and has collected 1 billion genetic data points, according to itswebsite. Brin and Google were early investors.

By contrast, 23Mofang is run out oftheChinese city of Chengdu, and Zhou, 36, is a computer science graduate who createdthecompany after becoming convinced Chinas next boom would be inthelife sciences sector. 23Mofang expects to have 700,000 customers bytheend of this year, a number he projects will at least double in 2020.

Thedivergence betweenthetwo countries andtheir regulation oftheindustry is just as palpable. Chinas race to dominate genetics has seen it push ethical envelopes, with scientistHe Jiankuisparking a global outcry last year by claiming to have editedthegenes of twin baby girls.Theexperiment, which He said madethem immune to HIV, put a spotlight on Chinas laissez-faire approach to regulating genetic science andthebusinesses that have sprung up around it.

When my reports came back, 23Mofangs analysis was much more ambitious than its American peer. Its results gauged how long I will live, diagnosed a high propensity for saggy skin (recommending I use products including Olay and Estee Lauder creams) and gave me an optimist not prone to mood swings a higher-than-average risk of developing bipolar disorder. 23andMe doesnt assess mental illness, which Gil McVean, a geneticist at Oxford University, says is highly influenced by both environmental and genetic factors.

Thefortune teller who pored over my palm told me I would live to be a very old woman. 23Mofang initially said I had a better-than-average chance of living to 95, before revisingtheresults to say 58% of clients hadthesame results as I did, making me not that special, and perhaps not that long-living.

When I ranthefinding pastEric Topol, a geneticist who foundedtheScripps Research Translational Institute in La Jolla, California, he laughed. Ninety-five years old?Theres no way to put a number on longevity, he said. Its a gimmick. Its so ridiculous.

Zhou saidtheaccuracy ofthelongevity analysis, based on a 2014 genetics paper, is not too bad, thoughthecompany plans to updatetheanalysis with research thats being undertaken on Chinese elderly.

But when it comes to disease,theresults of both companies showed howthescience of genetics, particularly attheconsumer level, is still a moving target.

Its All AbouttheData

After claiming I had a 48% greater risk thanthegeneral population of developing type 2 diabetes, both 23Mofang and 23andMethen revisedtheresults.

First, 23andMe cuttherisk figure from its analysis, posted in an online portal I accessed with a password.Theoverview analysis that I have an increased likelihood of developingthedisease never changed. But a few months later,thefigure was back, with a slightly different explanation: Based on data from 23andMe research participants, people of European descent with genetics like yourshave an estimated 48% chance of developing type 2 diabetes at some point between your current age and 80.

Shirley Wu, 23andMes director of health product, saidthecompany occasionally updates its analysis. My risk figure might have changed if I indicated my ethnicity and age, she said. I hadnt given any biographical details or filled out any surveys on 23andMes site.

Your risk estimates will likely change over time as science gets better and as we have more data, Wu said. We are layering in different non-genetic risk factors, and that potentially updates our estimates.

Algorithms and data underpintheanalysis of both companies, asthey do for other genetic testing firms, so it apparently isnt unusual forDNAanalysis to shift as more research and data into diseases becomeavailable. Still, I was confused.

I reached out to Topol, who said that 23andMes diabetes finding likely didnt apply to me sincethevast majority of people studied forthedisease are of European descent. Wu saidthe American company does have a predominantly European database but has increased efforts to gather data for other ethnicities as well.

23Mofang, meanwhile, also revised my diabetes risk to 26%. My genes hadnt changed, so why hadtheresults? CEO Zhou saidthecompany is constantly updating its research and datasets, and that may changetheanalysis. As time goes by,there will be fewer corrections and greater accuracy, he said.

For now, theres a possibility you can later get a result thats opposite oftheinitial analysis, said Zhou.

Additionally,theaccuracy of genetic analysis varies hugelydepending onthetraits and conditions tested because some are less genetically linkedthan others.

Zhou isnt deterred by criticism. He said 23Mofang employs big data and artificial intelligence to findthecorrelations to diseases without relying on scientists to figure it out.

While its impossible to get things 100% right,thecompanys accuracy will get better with more data, he said.

Ancestry Mystery

You might assume thatthetwo companies would offer similar analysis of my ancestry, which Ive long thought to be three-fourths Vietnamese and one-fourth Chinese (my paternal grandfather migrated from China as a young man). Born in Vietnam and raised intheUS, I now live in Hong Kong, a special administrative region of China.

23andMes analysis mirrored what I knew, but my ancestry according to 23Mofang? 63% Han Chinese, 22% Dai an ethnic group in southwestern China and 3% Uyghur. (It didnt pick up my Vietnam ancestry becausetheanalysis only compares my genetics to those of other Chinese, according tothecompany.)

That led me tothebig question in this grand experiment: How safe is my data afterthesetests?

Human Rights Watch said in 2017 that Chinese authorities collectedDNAsamples from millions of people in Xinjiang,thepredominately Muslim region thats home totheUyghur ethnic group. Chinas use of mass detention and surveillance intheregion has drawn international condemnation. What if Beijing compelled companies to relinquishdata on all clients with Uyghur ancestry? Couldthedetails of my Uyghur heritage fall into government hands and put me at risk of discrimination or extra scrutiny on visits to China?

23Mofangs response tothese questions didnt give me much solace. Regulations enacted in July gavethegovernment access to data held by genetics companies for national security, public health and social interest reasons.Thecompany respectsthelaw, said Zhou. Ifthelaw permitsthegovernments access tothedata, we will give it, he said.

Theauthorities havent made any requests for customer data yet, Zhou pointed out. Chinas State Council, which issuedtheregulations, andtheMinistry of Science & Technology didnt respond to requests for comment.

Over intheUS, 23andMe said it never shares customer data with law enforcement unlesstheres a legally valid requestsuch as a search warrant or written court order.Thecompany said its had seven government requests for data on 10 individual accounts since 2015 and has not turned over any individual customer data. It uses all legal measures to challenge such requests to protect customers privacy, said spokeswoman Christine Pai.

No Protection

New York Universitybioethics professorArt Caplansays privacy protections on genetic information are poor in most countries, including in the USand China.

I dont think anyone can say theyre going to protect you, he said. In China, its even easier for the government. The government retains the right to look.

23andMe appeals to potential customers with the lure of being able to make more informed decisions about your health, but after taking tests on both sides of the Pacific and realizing how malleable the data can be, as well as the myriad factors that determine diseases and conditions, I am left more skeptical than enlightened.

I gave away something more valuable than a vial of spit the keys to my identity. It could become a powerful tool in understanding disease and developing new medicines, but in the end its entrepreneurs like Zhou who will ultimately decide what to do with my genetic data. He plans to eventually look for commercial uses, like working with pharmaceutical companies to develop medicines for specific diseases.

We want to leverage the big database we are putting together on Chinese people, Zhou said. But first, we need to figure out how to do it ethically.

Excerpt from:
How do consumer DNA tests from the US and China stack up? - Abacus

Recommendation and review posted by Bethany Smith

Remember these two names, Lulu and Nana. They are twin girls born in China in October 2018, and we already know they are going to be famous.

They are the worlds first genetically-edited humans, and their progress through life will be monitored intensively by medical researchers over the coming years.

Dont doubt their future celebrity. Remember the headlines when Dolly the sheep arrived in 1996, the worlds first mammal cloned directly from an adult cell. Or how about Louise Brown, who made headlines in 1978 as the first baby to be conceived using invitro fertilisation (IVF) techniques.

Although the methods used to bring about these three births are light years apart, all three involve the delivery of an offspring using unorthodox methods.

All three were also controversial in their day. There was no end of condemnation and criticism about playing god and defying the laws of nature for the first two, but now IVF is commonplace for lots of medical reasons and is accepted as a standard medical practice. And news of another species successfully cloned in the lab would not make headlines today.

However, the genetic modification of the twins is a different matter, a genetic change introduced before birth to deliver a permanent alteration of their original genome that will be passed down from generation to generation.

Prof He Jiankui of the Southern University of Science and Technology carried out the genetic change needed to permanently modify the twins genome. He introduced a mutation that gave Lulu and Nana resistance to the Human Immunodeficiency Virus, the virus that causes Aids, and revealed his successful genetic modification one year ago this month.

The backlash was immediate and severe. There was international condemnation that China allowed experimentation on humans. Last January his university sacked him.

The scientific community also criticised the work as having crossed an important red line for genetics research, the reality that we still know too little about how even the smallest genetic change might have unexpected impacts downstream in other parts of the genome.

It was bad enough that a modification had taken place, but the modification was in the germline, the cells that bring about the next generation and the next and the next.

Perhaps the lure of notoriety proved too strong or the desire to be the first, but He Jiankui crossed that line, helped along the way through use of a gene-editing method known as CRISPR-Cas9.

This method emerged over several years, and has become the gene-cutting tool of choice because it allows very tight control over how a gene can be modified, added to or deleted.

When it came into widespread use the scientific community recognised immediately that controls on its application were necessary to prevent its use in human gene modification. This should have prevented He Jiankui from attempting such a daring and defiant experiment but it didnt, and Lulu and Nana were the result.

They were born healthy but now scientists will want to know whether there are unexpected or unwanted effects or other issues that arise. Already researchers have raised doubts about the gene modification that confers resistance to HIV, which means some level of susceptibility to the virus may remain.

The modified gene, known as CCR5, also has other roles in the body, and its modified actions may affect the lifespan of the twins. One large study involving 410,000 subjects showed that people with a similar mutated version of CCR5 were 20 per cent more likely to die before reaching the age of 78.

Despite these misgivings there is no doubt that CRISPR-Cas9 will in the future be used to modify the human genome in the battle against difficult diseases such as cancer and in genetic disorders caused by specific gene mutations. The tool is far too important to avoid its eventual use, or the use of some other similar but as yet unidentified gene-cutting method.

Similarly there is little doubt that online charlatans will offer promised but undeliverable cures using CRISPR-Cas9, as was the case with earlier technologies such as stem cells.

Lulu and Nana, meanwhile, will get on with living their lives, doing what babies do. Their names are pseudonyms in an attempt to conceal them from public view, but it is likely that we may learn their real names in the future.

Lulu and Nanas story came to mind in light of research published last week (November 21st) in the journal Cell about how attempting to create designer babies using other advanced technology could still remain a costly waste of time.

It involves choosing an embryo based on its potential to be tall or smarter than average, but accomplishing this via something like CRISPR-Cas9 is too far beyond our current abilities. Instead the international team of scientists set up a model to simulate one method called pre-implantation genetic testing.

This involves screening the genome for genes that have an association to a given trait, in this studys case intelligence and height, and giving them a score. The team found, however, that at best the top-scoring embryo might be expected to be 2.5cm taller than average and at best 2.5 IQ points above average.

Lurking behind all of this remains the most challenging of issues, the ethics surrounding the use of this technology.

As usual the ethics questions that should have been asked first are the ones obscured by the advance of these promising discoveries.

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Lulu and Nana are the result of a defiant experiment in human gene modification - The Irish Times

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SEATTLE The King County Public Health Department is working to determine the source of an outbreak ofE. colipotentially associated with four Evergreens Salad restaurants in Seattle.

Health officials said this strain of E. coli currently appears to be different from the strain causing the national outbreak of E. coli connected to romaine lettuce grown in the Salinas region of California.

Since Nov. 20, 2019, King County Health has learned of six people who have tested positive for E. coli after eating food at four different Evergreens Salad restaurants in King County. An additional person was diagnosed with E. coli and their infection is the same strain as the others, however, that person did not report eating at Evergreens.

The restaurants are located at:

All of those locations have an "Excellent" food safety inspection rating from the King County Health Department.

Health officials said between Nov. 21-25, Environmental Health investigators visited the Evergreens locations where the patients reported eating and they did not find any risk factors associated with the spread of E. coli, such as poor hand washing or improperly handling foods.

The Evergreens locations have also tossed all their romaine lettuce and extensively cleaned their cutlery and other items to cut down on any possible spread of the infection.

Evergreens released the following statement Tuesday evening, "Food safety is our top priority. We work every day using best practices so the food in our restaurants is safe and healthy. We have been in close contact with the health department and continue to cooperate fully to learn more about the source of the issue."

"People need to know if they ate at an Evergreens location and developed symptoms of E. coli, which can be diarrhea, which sometimes is bloody, nausea, abdominal cramping, that they should seek health care if their symptoms are ongoing," said King County Public Health officials.

Officials said this local outbreak could be the result of a contaminated product that was delivered and served at Evergreens. They also said some of the people who became sick after eating at Evergreens also said they ate raw vegetables, including leafy greens, from sources other than Evergreens in the days prior to their illness.

That means they could share a separate source for their illness, unrelated to Evergreens, officials said.

The King County Health Department has finished genetic testing on four of the seven local cases and officials said they did not match the genetic fingerprint of the strain connected to the national outbreak. However, health officials are still waiting for genetic testing from the other three cases.

King County health officials reported last week that one local man has been diagnosed with E. coli connected to romaine lettuce from the Salinas region.

However, again, officials do not suspect these new cases are connected to the national outbreak.

Evergreens setup a customer care number for guests: 877-394-4146.

RELATED: Don't wash your Thanksgiving turkey, food safety experts warn

RELATED: Dont eat romaine lettuce from Salinas, California, US officials say

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King County looks for source of E. coli outbreak possibly linked to Evergreens Salad chain - KING5.com

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MCELMO CANYON The apple orchard on Jude and Addie Schuenemeyers farm in a squiggle of a canyon in far southwest Colorado is a wild place. Turkeys gobble around on the hunt for bugs in native grasses that grow nearly as high as the gnarly limbs of the apple trees. Those trees are set hither and thither instead of lining up in typical tidy orchard rows. They bear apples that few fruit fans have likely heard of: Winter Banana, Blue Pearmain, Ben Davis and Esopus Spitzenburg.

This scraggly looking orchard is an important place in the country when it comes to preserving the historic outliers of a fruit famous for keeping the doctor away. In an orchard designed to be a water-saving pollinator sanctuary and a historic throwback, and in a nearby greenhouse filled with baby trees, the Schuenemeyers are preserving rare varieties of apples. They have hunted many of them down and grafted them from century old trees scattered and clinging to life around farms and yards in a corner of Colorado that once was world famous for its apples.

World famous is no exaggeration. Montezuma County apples took three of four gold medals in the St. Louis Worlds Fair in 1904. Two years later, they earned 101 of the 104 ribbons bestowed on apples at the Colorado State Fair.

The Schuenemeyers keyed in to this rich history in the early 2000s when they were running a nursery and hearing from old-timer customers nostalgic for apples they had known as kids. The Schuenemeyers began searching out historic trees, and Jude taught himself to graft so he could revive some of the old apple varieties.

Five years ago, they started the non-profit Montezuma Orchard Restoration Project to expand on their work. Since then, they have not only identified and saved hundreds of historic apple varieties from extinction, they have also sparked a renewed interest in apples. Apples are once again part of the Four Corners areas identity and its economic engine. Historic apples are a new point of pride in a region long known mainly for ancient ruins.

We have some of the rarest of the rare here in this area. We have thousands of apple trees that are 80 to 135 years old, Jude says as he picks his way through his own orchard followed by the curious turkeys and roughhousing dogs.

Through DNA testing, the Schuenemeyers have been able to identify nearly 500 varieties that had been planted in the southwest corner of Colorado prior to 1930.

That number is eyebrow-raising when compared to the fact that there are currently just 15 dominant apple varieties in American supermarket bins, including the Red Delicious, the Golden Delicious, the Granny Smith and more recently, the Honeycrisp.

Even more surprising; around the turn of the last century there were 17,000 varieties of apples growing across the United States, apples with intriguing names like Viberie, Black Oxford, Ashmeads Kernel and Duchess of Oldenburg. Currently, about 7,500 varieties remain worldwide.

The Schuenemeyers DNA testing shows that nearly 200 of the 500 varieties theyve found are considered rare. Another 100 or so are unique/unknown.

This local diversity is best seen in the Schuenemeyers greenhouse. In the past year they have grafted 1,600 twigs, also known as scions, from old trees onto new root stock. Hundreds of these little trees line up in buckets waiting to be replanted in demonstration orchards or in the backyards of buyers at the Montezuma Projects regular tree sales.

Each tree is tagged with a name, or with a number and letter code for the unknowns. When an unknown variety stands out for its notable appearance or taste, the Schuenemeyers bestow names like Carnation Salmon and Purple Mountain Majesty.

Jude moves through the greenhouse disseminating rapid-fire information about different varieties. He knows the growing season, the texture, the taste and the history of many of the trees. He points out particular favorites.

Isnt this guy gorgeous? he says as he riffles the reddish leaves of an unknown cultivar in the same loving way he fluffs the hair of his year-old daughter Hazel.

And look at this funny little guy. He points to a stubby tree that required numerous attempts at tongue-and-groove grafting to finally get a new start on life. He has no idea what it is just that it is no ordinary apple.

We are identifying the rarest of the rare here, he said.

Beyond the Schuenemeyers home and orchard, which is hidden behind heavy foliage in McElmo Canyon, it is easy to see spreading signs of their apple crusade.

The Four Corners area is now home to two cideries that take advantage of historic varieties of ugly, bitter apple varieties known as spitters. Spitters are especially suited for ciders. A third is planning to set up shop in an old juice factory in Dolores.

Id love to see the Four Corners become the Napa Valley of hard cider. I do think it has potential, says Martha Teal, who started Teal Cider at her historic T Lazy T Orchard near Dolores.

Sam Perry, an owner of Fenceline Cider in Mancos, has gone all in on the potential of apples. He planted 800 cider apple trees in the past three years and is planning to plant another 1,200 in the spring.

This area has been so reliant on cattle and hay for so long, he says. But I am hoping we are at a tipping point now for an apple renaissance.

There are glimpses of a renaissance where an orchard sprouts alongside a football field at Montezuma-Cortez Middle School. A new demonstration orchard has been planted in a park in the Town of Dolores a town that was once all orchard before homes and businesses moved in. That orchard, funded by a USDA Specialty Crop grant, will feature interactive materials so visitors can look up information on the histories and genetics of various varieties. An orchard of historic apples is also being planted near the small farming community of Yellow Jacket.

A historic Gold Medal orchard in McElmo Canyon that is being preserved in a partnership with The Nature Conservancy, is also a showpiece for the apple revival. It once sent ribbon-worthy apples to the Worlds Fair. An orchard also has been preserved near Hesperus, on a hillside at what was once a military fort and later an Indian boarding school.

The Montezuma Project has inserted apple education into local schools, organized apple-themed community socials, taught grafting classes and brought in mobile apple juicers. Local businesses have pitched in on the apple effort by making sustain-a-tree donations. They place their ads in an apple-of-the-month calendar alongside images of both brawny and winsome looking historic apple varieties.

The Montezuma project is also lending support to other apple preservation projects around the state and sharing genetic apple information with universities and other apple researchers around the country.

Nostalgia for historic apples has cropped up in other parts of Colorado where a diversity of apples once flourished areas around the North Fork Valley, Boulder, Caon City and the West End of Montrose County.

The nearly 3-year-old Boulder Apple Tree Project is doing many of the same identifying and preserving tasks as the Montezuma Project, but it has more of an academic focus because of its link with the University of Colorado. That project has no plans to try to revive an apple industry in that area, according to CU research assistant Amy Dunbar-Wallis.

CU students do in-the-field collections and in-the-lab genetic testing on historic apple varieties.

Last year the students tested samples from around 500 trees and identified about 90 individual varieties. This year they are testing another 300 trees and plan to reveal results at a Dec. 7 apple symposium that will include talks by the Schuenemeyers and by a USDA geneticist. It will also feature Katharine Suding, a CU plant ecologist who started the Boulder project after her son asked where did that come from? about a gnarly old apple tree in their backyard.

Dunbar-Wallis says the apple project has been popular with students as well as community members: A lot of it has to do with nostalgia. It connects people to the land and feeds that interest in knowing where their food comes from.

In a rural far-west area of Colorado stretching from Redvale to Paradox, a preservation effort called the Apple Core Project has been mapping, documenting and grafting scions from some of the 800 historic apple trees that have been identified there. Fifty historic varieties have been planted in a contoured demonstration orchard in the middle of Nucla, where apples used to come with similar bragging rights to Montezuma Countys.

In the early part of the last century Nucla-area apples like the Wolf River, the Yellow Bellflower, the Maidens Blush and the Seek No Further took 49 ribbons at a Colorado State Fair. A 1906 newspaper article trumpeted the tale of a magnificent apple grown in nearby Paradox and delivered to miners in Telluride. It had a 15-inch circumference.

Melanie Eggers, who founded the Apple Core Project with Jen Nelson four years ago, said they were inspired by the Montezuma Project. They borrowed some of the Schuenemeyers ideas for apple socials and educational events.

Eggers said it is not unusual to have townspeople stop by the orchard in Nucla to share memories of the old orchards and thank them for reviving a bit of the apple heyday.

One older woman cried and said, This gives me hope for our future generations, Eggers recalls. It turns out this is very meaningful for our community; more so than we ever thought.

All these apple-revival areas suffered from the same historical forces weather, politics and industrialized farming that sidelined so many varieties of apples.

The many varieties came to Colorado with pioneers who planted so many types of apples for good reason. Because different varieties ripened at different times and had different shelf lives, they yielded a year-around supply of fruit. Families could also handle the staggered harvests without having to hire outside crews.

Some of the trees were chosen to yield the spitters for hard ciders. Because they had no use other than to make booze, most fell to the axes of FBI agents during Prohibition.

Washington state played a big part in killing off the cornucopia of apple varieties in Colorado. Growers in Washington, a less challenging place weather-wise to grow apples, embraced the idea of an apple monoculture that could yield huge amounts of long shelf life fruit that could be shipped across the country by rail. Old varieties were torn out to make way for the Red Delicious which, at its height of popularity, accounted for 80% of all apples grown in the country.

That shift turned apple farming into the apple industry. Colorado tried to join that industry to compete with Washington. Crops were rejiggered to favor uniform, blemish-free globes. The Pitts Bitters, the Knot Heads and the Wine Kissed didnt stand a chance. But Colorado was never able to catch up, and many orchards were ripped out to make way for more profitable crops.

The current effort to identify and preserve these nearly lost varieties is happening against the backdrop of another Washington-fueled apple blitz the fruit equivalent of a new iPhone release.

Washington has developed a new variety called the Cosmic Crisp, set to hit stores on Dec. 1. It got its name from the tiny yellow dots that look like distant stars on a red background. Growers there have the exclusive right to grow and sell the apple for the next decade.

This goes against the grain for apple variety preservers like the Schuenemeyers, who devote their efforts to getting as many apple varieties back out into the world as possible.

History and ever-changing consumer demand show that the favorite apple of today may become scorned tomorrow, and the apple scorned today may be sought after in the future, Addie says. Therefore, there will always be a place for fruit preservation efforts.

That doesnt mean the Schuenemeyers are averse to identifying a Cosmic Crisp-like star of historic apples. They have been searching for years for a once prized apple called the Colorado Orange that was thought to be extinct. They thought they found it on an old tree near Florence several years go, but DNA tests showed it was something else.

The Schuenemeyers now have another suspected Colorado Orange that DNA tests have deemed an unknown/unique. If it matches up to a wax apple in a long-forgotten collection at Colorado State University, they will be a step closer to declaring they have found the venerable Colorado Orange. And they will have saved a variety on its last leg; the tree is down to a single limb. The Schuenemeyers have succeeded in grafting 50 scions from it so far.

Their eyes light up when they set a few of those maybe Colorado Oranges on a stump at their orchard. The sun lights up the apples blush. The Schuenemeyers could be looking reverently at a piece of fine, valuable art.

I think we have inspired people to see that old trees have value, Addie says.

Jude simply nods, a dreamy smile on his face and a faraway look in his eyes.

This reporting is made possible by our members. You can directly support independent watchdog journalism in Colorado for as little as $5 a month. Start here: coloradosun.com/join

Go here to read the rest:
An apple revival near Four Corners is restoring hundreds of historic fruits and the local ag economy - The Colorado Sun

Recommendation and review posted by Bethany Smith

By Citizen Staff | on November 28, 2019

PartnerMD will open its thrd Richmond-area location at GreenGate in Henricos Far West End in the spring of 2020, company officials announced this week.

The location will be the companys seventh overall and was prompted by what officials termed significant individual and corporate customer growth over the past two years.

PartnerMD describes itself as a concierge primary care and executive health practice.

Were very excited to offer a new, convenient location for patients in Richmonds far West End, PartnerMD CEO Zack Smith said. Our rapid growth and expansion of physicians and services has pushed the limits of our existing offices at Reynolds Crossing and Midlothian, and were thrilled to better serve both the residents and companies in this fast-growing part of our hometown market.

PartnerMD recently added two new physicians locally: Dr. Elizabeth Bigelow and Dr. Steven Bishop. Bigelow, a family physician, joined in October from the Hunter Holmes McGuire VA Medical Center and will transition to GreenGate upon opening. Bishop, an internist who starts in December from VCU Health, will see patients at Reynolds Crossing and serve as director of wellness at PartnerMD to continue the growth of the companys disease-prevention programs around such holistic health topics as weight loss, diabetes, sleep, and stress.

PartnerMD expanded its wellness offerings this year through a new genetics partnership with VCU Health. Through the partnership, patients have onsite access at PartnerMD offices to the advanced and personalized genetics counseling, as well as genetic testing that uses VCU Healths CLIA-certified laboratories.

Here is the original post:
PartnerMD's third location to open at GreenGate in Henrico - Henrico Citizen

Recommendation and review posted by Bethany Smith

It may finally be a happy Christmas for a brave toddler recovering from a rare combination of cancers after pioneering stem cell treatment.

Amelia Topa, who celebrated her second birthday yesterday, is looking forward to spending the festive period with her family at home in Turriff.

Her parents Kerri Paton, 23, and Igor Topa, 24, were told that raised purple spots across Amelias body could be a sign of something seriously wrong when she was born at Dr Grays Hospital in Elgin.

Amelia was soon after diagnosed with a mix of two types of leukemia acute lymphoblastic leukaemia and acute myeloid leukaemia and spent Christmas in hospital.

Miss Paton said: Its rare enough to be born with leukaemia but to be born with a mix of two kinds is almost unheard of.

Doctors gave Amelia a bone marrow transplant using stem cells donated by a managed between 16 and 30.

The treatment worked and, by the following autumn, she was home and awaiting the arrival of her baby brother.

But tragedy struck when Amelias grandmother, Angela McNabb, died suddenly from heart failure aged 48 the day before Amelias birthday.

Miss Paton said: My mum was my best friend, she was everything to me.

Mum absolutely loved Amelia and was so close to her.

My major source of support was gone and I hadnt even had the chance to say goodbye. I couldnt believe it. It was so unfair. Last Christmas was heartbreaking.

Things went from bad to worse for the family in February, when tests showed that Amelias cancer had returned.

After intense chemotherapy she was given a second transplant using stem cells from umbilical cord blood flown specially from America at the end of June.

That procedure was a success and the toddler has entered remission.

Having spent Christmas in 2017 in hospital, and in mourning last year, Amelias parents are now looking forward to a happy festive season.

Miss Paton said: Amelia soared through the transplant and shes doing really well now,I couldnt be prouder.

I hope Amelias story will help other families going through cancer there is a light at the end of the tunnel.

Amelia has now been selected to receive the first Cancer Research UK children and young people star award in recognition of the courage she showed since being diagnosed.

The award, supported by TK Maxx, is open to all people under 18s who currently have cancer or who have been treated in the last five years with every child being awarded a trophy, TK Maxx gift card, t-shirt and certificate signed by the likes of Nanny McPhee star Dame Emma Thompson.

Spokeswoman for the charity, Lisa Adams, said: We know that a cancer diagnosis is devastating at any age, but that it can be particularly difficult for a child or young person and their families.

Thats why were calling on families across Scotland to nominate inspirational youngsters for an award so that we can recognise their incredible courage.

Nominations can be made online at cruk.org/childrenandyoungpeople

Visit link:
North-east toddler overcomes cancer after pioneering stem cell treatment - Press and Journal

Recommendation and review posted by Bethany Smith

Regenerative Medicine and Stem Cell technologies... All Hype? Or The Future of Anti-Aging?

We have entered into the rapidly evolving age of Regenerative Medicine, using breakthroughs in cell to cell communication to reset your body's natural ability to heal and repair itself. Advances in Stem Cell technology race forward at an ever increasing rate as people just like you are demanding non-surgical alternatives to the multiple degenerative conditions of aging and inflammation.

If you are asking What is a Stem Cell?

Think of them as simply the master cells of rebuilding the body. They are the building blocks of our genetic code and cellular programming that coordinate healing through bio-signaling to restore our innate capacity of repair & regeneration.

Regenerative Medicine is the vanguard of 21st century health According to Journal: American College of Cardiology

Regenerative Aesthetics is a new field of regenerative medicine that aims to restore and renew the body at the cellular level, dramatically reversing the sands of time and maintaining an aesthetically desirable youthful appearance.

What we really now know is that men and women all over the world want their hair back, they want their sexual organs to work and they want to look and feel their best into their later years.

This brings us to the 3 Regenerative Therapies you should know about.

The first and most widely known of these regenerative therapies is Platelet Rich Plasma or PRP which utilizes your own Blood Plasma and Platelets in a concentrated form to activate the healing cascade. Recruiting your own innate Stem Cells for accelerated wound healing and tissue regeneration.

Known as Liquid Gold, your platelets and plasma have been shown to rejuvenate the

Unfortunately, we are finding clinically that not all PRP is created equally. In fact, some people have a very low concentration of these regenerative growth factors or a high amount of inflammatory cytokines resulting in inconsistencies from person to person, session to session. As PRP continues to get more popular in the mainstream, we find it important to share some of the newer, more optimal Regenerative Technologies that have been emerging.

Which leads us to... The second regenerative therapy you should know about.

Stem Cell Growth factors & Cytokines. Sourced from Bone Marrow Mesenchymal Stem Cells (MSC's) however, they do not contain any actual stem cells or DNA. Growth factors are naturally occurring proteins in your body that regulate cellular growth, healing, proliferation and differentiation under controlled conditions and play a role in cellular communication. They are master bio-signals acting as command and control over your body's natural healing processes and modulation of inflammation.

It has been shown that cells in aging skin generate less growth factors than cells in youthful skin. For example, by the time you are 50, on average 4% of these regenerative bone marrow MSCs are in circulation compared to what you had when you were in your teens. Hence, we age because we damage faster than we repair in our later years.

By simply adding concentrated MSC Growth factors & Cytokines to our Regenerative Therapies we can consistently improve hair loss, skin rejuvenation, collagen growth, sexual organ function, and more. We know for a fact that daily use of skin care products containing stable growth factors and cytokines help reduce the appearance of fine lines & wrinkles and improve skin tone & texture.

Lastly, and most importantly is the latest frontier and the 3rd regenerative therapy you should know about.

The Future of Regenerative Medicine... Known as Stem Cell Exosomes. Science is showing us that the optimal way to provide true stem cell therapy is to directly provide the cell bio-signals in high concentrations. After all, the signaling is what we really require to regenerate a normal healthy physiology.

Exosomes are regarded as the purest form of cellular therapy available today, providing a safe and anti-inflammatory environment for healing and repairing.

The AABB recently reported that up to one in three people in the U.S. could benefit from regenerative medicine.

At Rejuvenate 528 Regenerative Aesthetics Medical Spa, we can include exosomes for enhanced wellness to the majority of our Regenerative Aesthetic Services. This can benefit your overall health and vitality as this is reversing challenges of Inflammation!

Beauty radiates and vibrates at different frequencies in everyone I see! We love to uplift and Rejuvenate both the inner vitality and the outer Radiance of all of our patients and clients. They come for the personal attention and integrative approach using regenerative medicine modalities with ancient technologies. PA Sheri Suiter

Tap into your own healing potential with these types of bio-hacking technologies to enhance your regenerative potential and get the results you truly desire. Live longer, stronger and younger.

Book a consultation for the following Regenerative procedures:

*Medical Microneedling*Vampire Facial*Liquid Facelift*Breastlift*O-Shot*P-Shot*Penile Enhancement*Hair Restoration*Stretch mark & scar repair*Hand Rejuvenation*Joint Inflammation*Overall Vitality & Wellness

Sheri Suiter CLT, MS, PA-C, Founder of Rejuvenate 528 Regenerative Aesthetics Medical Spa in Sarasota, FLRejuvenate528.com

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Discover The Latest Frontier in Anti-aging Medicine and 3 Regenerative Therapies You Should Know... - YourObserver.com

Recommendation and review posted by Bethany Smith

A TODDLER born with two types of leukaemia will spend Christmas at home after recovering from a second stem cell transplant.

Amelia Topa, who turned two on Wednesday, was donated cells harvested from a newborn babys umbilical cord blood flown in from the US.

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The brave youngster is now in remission and is preparing to enjoy the festive season with relieved parents Kerri Paton, 23, and Igor Topa, 24, in Turriff, Aberdeenshire.

Recalling Junes life-saving op, mum Kerri said: Amelia soared through the transplant and shes doing really well. I couldnt be prouder.

Its rare enough to be born with leukaemia but to be born with a mix of two kinds is almost unheard of.

Worried medics alerted Amelias parents to raised purple spots on her body shortly after she was born at Dr Grays Hospital, Elgin.

She was diagnosed with acute lymphoblastic leukaemia and acute myeloid leukaemia and spent Christmas 2017 in hospital.

The tot was given a bone marrow transplant six months later using stem cells donated by a man aged between 16 and 30.

The treatment worked but tests showed Amelias cancer had returned in February.

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She endured gruelling chemo before her second transplant in the summer.

Kerri added: I hope Amelias story will help other families going through cancer. There is a light at the end of the tunnel.

Amelia has now been chosen to receive Cancer Research UKs first Children & Young People Star Award.

The prize, backed by TK Maxx and stars including Dame Emma Thompson, is open to under-18s battling cancer or who have been treated in the last five years.

The charitys Lisa Adams said: Were calling on families to nominate inspirational youngsters so we can recognise their courage.

VISIT cruk.org/childrenand youngpeople to nominate.

scottish-sun@the-sun.co.uk

We pay for your stories and videos! Do you have a story or video for The Scottish Sun? Email us atscoop@thesun.co.ukor call 0141 420 5300

Originally posted here:
Brave Scots tot born with two types of leukaemia to spend Christmas at home after second stem cell transplant - The Scottish Sun

Recommendation and review posted by Bethany Smith

Hematopoietic stem cells (HSCs) can be used to better understand and treat blood-based diseases. Stem cell research in the model organism zebrafish is well-studied in the developmental stage, but is limited in the adult stage because HSCs are difficult to purify in this species. Researchers at Kanazawa University and their collaborators have developed a new purification scheme that allows HSCs to be purified from adult zebrafish kidneys, potentially opening new possibilities for stem cell research.

Kanazawa, Japan Hematopoietic stem cells (HSCs) are multipotent cells that can develop into every type of blood cell in the body. They can also be used in medical research to understand and treat blood-based diseases. Zebrafish (Danio rerio) are used to study HSCs, particularly in the field of developmental biology, but the research in the adult animal is often limited because stem cells are difficult to purify in this species. Researchers at Kanazawa University and their collaborators now describe a purification scheme that allows these elusive zebrafish HSCs to be collected.

Zebrafish are a great system to study how hematopoietic cells function in normal development and their role in disease, says lead researcher Isao Kobayashi. Much of their biology mirrors what we see in humans, and with zebrafish theres the added benefit of having quite a few experimental tools at our fingertips, including live cell imaging and comparative analysis among vertebrates. Unfortunately, its proven challenging to effectively isolate HSCs from this species, and this has been a major impediment to the field.

HSCs are highly abundant in the kidneys of adult zebrafish (unlike in humans, where HSCs are found in bone marrow). The challenge is separating them from other cells found in kidneys. Cell separation usually involves a purification technique called flow cytometry, where cells are sent in single file through a tube and hit with a laser beam. The machine (a flow cytometer) then sorts the cells based on how they reflect or scatter light.

In the study, published in Scientific Reports, the researchers created a strain of zebrafish that makes two light-emitting proteins, one green (Green Fluorescent Protein, GFP) and one red (mCherry), that can be sensed and sorted by a flow cytometer. Each fluorescent protein in this zebrafish strain was regulated by the genes related with blood cells, but the cells having both fluorescent proteins were limited in HSCs. By color coding the cells with two distinct blood cell markers, the team was able to purify cells that show hallmark signs stemness like the ability to self-propagate and differentiate into other types of blood cells.

So, what might the successful isolation of HSCs in zebrafish mean for the field of stem cell research?

When HSCs were finally purified in mice, the research community learned an enormous amount about how and where stem cells self-renew and differentiate to form blood cells, says co-author Mao Kondo. Were very hopeful that this might spur a similar proliferation of research in zebrafish. In addition to some experimental advantages in zebrafish, we found that zebrafish HSCs share many key genes in common with HSCs in mammals. This suggests that mechanistic discoveries in zebrafish could have direct implications for understanding blood diseases in humans and for developing new medical treatments.

Figure.

Hematopoietic stem cells can be isolated as gata2a:GFP+ runx1:mCherry+ (gata2a+ runx1+) cells in the zebrafish kidney by flow cytometry (left panels). Transplantation assays confirmed the hematopoietic potential of gata2a+ runx1+cells (right panels).

Article

Enrichment of hematopoietic stem/progenitor cells in the zebrafish kidney

Journal: Scientific Reports

Authors: Isao Kobayashi, Mao Kondo, Shiori Yamamori, Jingjing Kobayashi-Sun, Makoto Taniguchi, Kaori Kanemaru, Fumihiko Katakura & David Traver

DOI: 10.1038/s41598-019-50672-5

Funder

This work was supported in part by Grant-in-Aid for Young Scientists (B) from the Japan Society for the Promotion of Science (17K15393).

Link:
Researchers Find a Way to Collect Elusive Blood Stem Cells from Zebrafish - Mirage News

Recommendation and review posted by Bethany Smith

The parents of a baby girl were left heartbroken after she was diagnosed with two types of blood cancer.

Amelia Topa, who turns two today, is in remission from leukaemia after having a stem cell transplant using a newborn babys umbilical cord blood which was specially flown in from America.

Now doing well, Amelia has received a Cancer Research UK for Children & Young People Star Award, supported by TK Maxx, in recognition of the remarkable courage she demonstrated since being diagnosed with cancer.

Amelias parents, Kerri Paton, 23, and Igor Topa, 24, of Turriff, Aberdeenshire are hugely proud of their little girl.

Kerri said: Anyone who meets Amelia would agree that shes a star.

READ MORE - Scottish actor Gray O'Brien reveals he has been treated for stage four cancer

Being told your child has cancer is the worst sentence any parent could ever hear. I felt mad at first that someone so tiny should have to go through this horrible disease. But Amelia has been a little fighter from the day she was born. I have felt amazed by her strength and lucky to have good support from friends, family and hospital staff.

We will forever be grateful to a family in America well probably never get a chance to meet. The stem cells from America looked just like a small bag of blood but they had the power to make Amelia well again.

Around 140 children are diagnosed with cancer in Scotland every year.

Mum Kerri recalls vividly the moment their lives were turned upside down when only hours after Amelia was born on November 27 2017 medics at Dr Grays Hospital in Elgin explained that raised purple spots across Amelias body could be a sign of something seriously wrong.

Following tests, on December 14 2017 Amelia was diagnosed with leukaemia. Unusually, doctors diagnosed Amelia a mix of two types, acute lymphoblastic leukaemia and acute myeloid leukaemia. The family were transferred to the Queen Elizabeth University Hospital in Glasgow the following day.

READ MORE - Gary Rhodes: Celebrity chef dies suddenly aged 59

Kerri said: It didnt hit me properly until I walked out of the room and then I started crying, a lot.

Its rare enough to be born with leukaemia but to be born with a mix of two kinds is almost unheard of. We were looked after by the hugely experienced Professor Brenda Gibson. It helped to know we had the best oncology doctor on our side.

Amelia spent her first Christmas in hospital as the first of four rounds of chemotherapy treatment started. By spring, the family were advised that Amelias best chance of survival was a bone marrow transplant using stem cells. A match was found and the transplant went ahead on June 28 2018. The family were told was that the stem cells had been donated from a man aged between 16 and 30.

Amelia recovered well and tests showed that the transplant had worked. By autumn last year Amelia was well enough to go home and the family slowly settled back in to life in Aberdeenshire. And after a difficult year, it was a boost when Kerri discovered she was pregnant again.

Oscar was the first baby to be born in the new maternity unit in Aberdeen when he arrived on October 30 2018. Now Amelia was big sister to Oscar, Kerri dared to hope they could settle in to an ordinary family life.

But tragedy struck again. Kerris mum, Angela McNabb who had stood by the family every step of the way suddenly died from heart failure aged 48- just the day before Amelias birthday.

Kerri said: My mum was my best friend, she was everything to me.

Mum absolutely loved Amelia and was so close to her. My major source of support was gone and I hadnt even had the chance to say goodbye. I couldnt believe it. It was so unfair. Last Christmas was heartbreaking.

And it was a hammerblow on February 11 this year when tests showed that Amelias cancer had come back. Doctors were uncertain at first whether anything else could be done but they suggested a second stem cell transplant, this time using stem cells from umbilical cord blood.

Amelia had intense chemotherapy in an isolation room before she was ready for the transplant at the Queen Elizabeth University Hospital in Glasgow. The transplant went ahead on June 28 this year- exactly a year after the first transplant.

Kerri said: They had to fly the umbilical cord blood over from America.

Doctors explained to us that this was the best option to keep the leukaemia away. Amelia soared through the transplant and shes doing really well now. Were finally looking forward to a happy Christmas as a family and I couldnt be prouder. I hope Amelias story will help other families going through cancer. There is a light at the end of the tunnel.

The Cancer Research UK for Children & Young People Star Awards, supported by TK Maxx, are open to all under-18s who currently have cancer or have been treated for the disease in the last five years. There is no judging panel because every child diagnosed with cancer deserves special recognition. Everyone nominated receives a trophy, 50 TK Maxx gift card, t-shirt and a certificate signed by a host of famous faces, including Nanny McPhee and Last Christmas star Dame Emma Thompson, This Mornings Dr Ranj and childrens favourite entertainer Mister Maker. Their siblings also receive a certificate.

Now they are encouraging families across Scotland to nominate their stars for the honour in the run up to Christmas.

Lisa Adams, spokeswoman for Cancer Research UK for Children & Young People in Scotland, said: Our Star Awards, supported by TK Maxx, shine an important light on children and young people with cancer.

We know that a cancer diagnosis is devastating at any age, but that it can be particularly difficult for a child or young person and their families. Thats why were calling on families across Scotland to nominate inspirational youngsters for an award so that we can recognise their incredible courage.

The Cancer Research UK for Children & Young People Star Awards are supported by TK Maxx, the biggest corporate supporter of the charitys research in to childrens and young peoples cancers. Since the partnership began, the retailer has raised over 34 million for research in to these cancers to help more children and young people survive cancer.

Originally posted here:
Scottish baby spent first Christmas in hospital after she was diagnosed with two types of cancer - Scotland on Sunday

Recommendation and review posted by Bethany Smith

Hailie Hyman holds her daughter Maci, 1, before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

GREENVILLE, S.C.Hailie and Treylin Hyman saw the bruising on their baby girls leg as a sign that the active 1-year-old was learning to walk.

But as a blood test would later reveal, little Maci was actually suffering from an extremely rare blood cancer that threatened her life without a risky treatment - atreatmentalmost as dangerous as the disease.

In the beginning, it was very scary, Hailie Hyman told The Greenville News.

I couldnt think of anything but the bad things, she confessed. It was all about the statistics. And the statistics arent good.

Terrifying months followed the diagnosis, punctuated by one critical complication after another, leaving the Boiling Springs couple to wonder if Maci would survive.

Somehow, though, the blue-eyed toddler pulled through.And now her family is looking forward to a special Thanksgiving with much to be grateful for.

Alyssa Carson is 18 and has a pilot's license: She wants to be in the crew that colonizes Mars

The Hymans journey began last February atMacis 1-year-old well-child checkup.

We had no idea anything was wrong, her mom said.But they did a routine (blood test) and a couple of hours later, we got a call saying her platelets were very low.

The Hymans were referred to a hematologist who found other abnormalities in Macis blood and scheduled a bone marrow biopsy to investigate further.

Hailie Hyman holds her daughter Maci, 1, before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

During the procedure, the child suffered an aneurysm in an artery and went into cardiac arrest. The team performed CPR on her for 20 minutes before she was stabilized, her mom said.

Later, in the pediatric intensive care unit, she suffered internal bleeding, too.

It was really hard, she said. There were many nights that I would just pray and pray and pray.

Initially believing Maci had leukemia, doctors subsequently determined she had myelodysplastic syndrome, or MDS.

The condition occurs when abnormal cells in the bone marrow leave the patient unable to make enough blood, according to the American Cancer Society.

Its rare, afflicting as few 10,000 Americans a year, though the actual number is unknown.

Maci Hyman, 1, interacts with hospital staff before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

In children, its rarer still. Most people arediagnosed in their 70s.

We were told that just four out of 1 million children get it every year, Hailie Hyman said.

That made the diagnosis elusive at first, said Dr. Nichole Bryant, a pediatric hematologist-oncologist with Prisma Health-Upstate, formerly Greenville Health System.

Shes the only one Ive seen in my career, she said.

Maci had to have regular blood transfusions, antibiotics and other medications to fight the MDS, Bryant said. But the only hope for a cure was a stem cell transplant at the Medical University of South Carolina in Charleston.

When they said that was the only treatment plan for MDS, I of course went to Google, Hailie Hyman said. I read about transplant patients and ...all the complications. It was terrifying. But no matter how many bad things I saw, we had to do it. There is no other option.

The transplantis extremely risky.

Hailie Hyman looks at a fish tank with her daughter Maci, 1, before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

First, high doses of chemotherapy are given to destroy the diseased bone marrow, leaving the patient without an immune system, so fighting infections becomes a challenge. Then healthy donor marrow is infused.

Its also fraught with potentially life-threatening complications, including graft vs. host disease, which occurs when immune cells from the donor attack the patients body, Bryant said. Other complications include permanent kidney damage and gastrointestinal problems.

They have to go to hell and back, she said. But its the only option for long-term survival.

Maci had a really rough start, suffering lots and lots and lots of complications, Bryant said.

Her kidneys failed, so she wound up on dialysis. When she couldnt breathe on her own, she was put on a ventilator. And because she couldnt eat, she had to be tube fed.

Hailie Hyman looks at a fish tank with her daughter Maci, 1, before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

She had blistering sores in her mouth and throughout her GI tract, her mom said. Because her liver wasnt functioning properly, her abdomen filled up with fluid that had to be drained. She was bleeding so profusely in her lungs that one of them collapsed.

Maci, who was sedated through much of it, was put on full life support, she said.

That night we almost lost her, her mom said. We were in the hallway crying our eyes out. We didnt know what do to or think. It was pretty scary for a while.

Somehow, Maci made it.

There were so many times during her first months that it seemed like she would not survive, Bryant said. So the fact that she is here ... is really a miracle.

Macis family found an unrelated donor through the National Marrow Donor Program, enlisting hundreds of other people to join the registry in the process, Bryant said.

Nichole Bryant, M.D.(Photo: Provided)

It was an important part of their journey that maybe didnt directly benefit Maci, she said. But if everybody did that, we wouldnt have difficulty finding a donor for anybody.

Doctors have no explanation for why Maci got MDS. She didnt carry the genetic mutation for it and there is no family history.

She is a rare child - and not in a good way, her mom said, adding,Youve got to laugh sometimes or youre going to cry.

A dying man wanted one last beer with his sons: The moment resonated with thousands

Maci was admitted to MUSC on June 2 and released on Oct. 14.

The Hymans, both 22, spent the entire time in Charlestonwhile Hailies mom cared for their older daughter, Athena, now 2.

Treylins employer held his welding job open for him. And other friends and family members did what they could to help.

We had many, many people very generously donate to us to cover expenses at home and living expenses where we were, Hailie Hyman said.

We are thankful for everyone who helped us through it the cards, the gifts, the donations. Every single cent is greatly appreciated.

Maci's doing well, but recovery from a transplant can take months to years, Bryant said.

Her kidneys are functioning again so she was able to come off dialysis. But she still must take many medications, including anti-rejection drugs that suppress her immune system and leaveher at risk for infection. And she still must be tube fed.

She is miles ahead of where she was two months ago, Bryant said. But she still has a long way to go. Its a long, long road.

Macis mom says she can be up and playing one day and flopped over on the couch another. She still experiences a lot of nausea and vomiting, but is doing well compared to where she was.

Hailie Hyman pulls her daughter Maci, 1, in a wagon in the hallway before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

So as the nation pauses to give thanks this Thanksgiving, she says the family will be countingtheir many blessings family andfriends, Gods mercy, andthe doctors and nurses who saved Macis life.

She has battled a lot and overcome a lot, she said. I have no doubt she will be able to get through.

Want to know more about becoming a marrow donor? Go to bethematch.org.

Follow Liv Osby on Twitter:@livgnews

104-year-old woman bags first buck: 'Never underestimate the power of our senior citizens'

Indiana hospital sued: More than 1,000 patients potentially exposed to HIV or hepatitis

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South Carolina toddler survives rare cancer and the risky procedure used to treat it - USA TODAY

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Appiah rang Leukaemia Cares helpline from the point of diagnosis until well after the end of her daughters life. Sometimes Id call them as a means of support, she says, when things got really rough, when her medications were really powerful, and the chemo made her so unwell. She rang when she had panic attacks; an NHS psychologist had told her that these were likely, and that she should breathe into a brown paper bag, but Appiah found speaking to a person more soothing.

With a laugh, Appiah notes that shed ring the helpline at other times, too: Sometimes Id be out with Imogin, and shed be in the pram, being naughty, and all of my patience was going down the drain, and Id phone Leukaemia Cares nurses, and say: Look, Im feeling so depressed, my daughters shouting, I dont know what to do!

But I might also say: Nurse, Im actually feeling good today.

Appiah says the support of an independent person was invaluable: When your child is so ill, you need to speak to someone who doesnt know your name you need an outsider you can unload to. I didnt want anyone thinking: Here Sheila comes again!

You become self-conscious about your situation and dont want to be a burden on your friends and family. With the helpline, you wont be judged: they just listen. You get it out of your system and then go do the shopping at Sainsburys.

When Imogin was well, shed go to school. But she also spent weeks at a time in isolation in St Georges Hospital, with her mother by her side. Once, she had a bad reaction to a medication and went into cardiac arrest. She was crying and saying, Please, please! and they were giving her all sorts of medicine. The doctors were battling to keep her stable and I dived into the bed with her and told her: Youre going to be OK. I lay down with her and I started singing with her. And then, once she stabilised, she said: Now can I watch High School Musical?"

Appiah shakes her head, laughing: Thats what she was like: I was on thedoor of death, but I have something else planned. I want to watch my video and none of you are going to stop me!

Charities sent the pair to Disneyland Paris twice. The first time was fantastic, says Appiah, the second time Imogin was in and out of consciousness. But they said we should go, to make memories, Appiah explains. Imogin got to be a celebrity for a day and went to Hamleys in a limousine to get anything she wanted.

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'My daughter's death took me to the darkest place, but I've learned it's possible to come back' - Telegraph.co.uk

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Angie Grice is a such huge Clemson football fan that in 20 years shes rarely missed a home game or any of the Tigers-Gamecocks match-ups.

At her tailgate parties, the plates, the tablecloth and even a rug are orange.

Angie Grice gets a visit from the Clemson Tigers mascot during her three months in the hospital.(Photo: Bon Secours St. Francis Health System)

But for this years annual Thanksgiving weekend face-off between Clemson and USC, shell be watching from home.

Diagnosed with aplastic anemia in May, the Simpsonville woman spent three months in the hospital and is still too sick to cheer her beloved Tigersfromthe stadium. Instead, she hopes to have a few friends over to catch the gameon TV.

Ive liked Clemson forever," she told The Greenville News.

"Im missing the game this yearand Im sad about that, she said.But its OK. At least Im able to watch it.

Grice, 52, first realized something was wrong in August 2018 when she suddenly had trouble crossing the parking lot from her car to her job as a physical therapy assistant.

I was very short of breath, she recalls. It would take me a long time to do anything. I just couldnt breathe.

She saw her family doctor, who sent her to Bon Secours St. Francis Health System when her blood work wassuspicious.

Angie Grice at Clemson University(Photo: Angie Grice)

An initial bone marrow biopsy was negative.But a second revealedaplastic anemia,which prevents the bone marrow frommakingenough new blood cells for the body to function normally, according to the National Institutes of Health.

The condition is so rare it strikes only 600 to 900 Americansa year, according to the The Aplastic Anemia and MDS International Foundation.

Symptoms include fatigue, weakness, dizziness, shortness of breath, infections, and easy bruising or bleeding,the NIHreports.The cause can bethe bodys own immune system attackingthe bone marrow, heredity, some drugs, and certain toxins likepesticides and benzene.

When St. Francis hematologist Dr. Fahd Quddus first saw her, Grices platelet level was 8,000 compared to a normal of 150,000.

Whenever you drop below 20,000, youre at risk of significant, life-threatening bleeding, he said. She also had significant anemia. And her white cells were also very low.

She was started on immunosuppressive medication and other drugs in combination with blood transfusions. But sadly, he said, she suffered multiple infections, fevers and a mild stroke, requiring her to stay in the hospital.

Dr. Fahd Quddus(Photo: Bon Secours St. Francis Health System)

For a few weeks, it was touch and go, Quddus said. She was very sick.

Grice'sblood counts eventually rebounded and though shes now out of the hospital, shestill needsregulartransfusions.

She's wellenough to begin a new treatment, he said, butnot yet strong enoughfor a stem celltransplant.

Theresstill a long road to recovery, Quddussaid. But she always looks at it half full. And thats a good thing because people who stay positive can do better.

No longer able to work because of the weakness and danger of infection, Gricesays shes doing OK thanks tofamily and friends.

Angie Grice at a Clemson game(Photo: Angie Grice)

My mom and dad and sister help, she says. And I am truly blessed with a lot of friends who help.

In years past, Grice and her friends arrived at the stadiumseveral hours before kick-off, spending 10 to 12 hours thereon game days.

Inside their orange tent, they set up a coupleTVs to watch other games before and after the Clemson game. There was always plenty ofgood food,smack talk and Tigersmerchandise.

Were a little over the top, she says. But its fun.

During her grueling three-monthhospital stay, it was a visit from the Clemson Tigers mascot that lifted her spirits.

One of Angie Grice's many Clemson decorations(Photo: Angie Grice)

While watching from home wont be as exciting, Grice says shes going to make the best of it. And when asked whos going to win this years game, sheexclaims, Clemson, of course!

If you ask Carolina, they will say they are, she adds with a chuckle. But theyre delusional. Were going to win this year.

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Despite illness, this Clemson fan will be tuned in for the Tigers-Gamecocks game - Greenville News

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Gavin McNaney, who passed away two years ago.

Translink are hosting a blood stem cell registration event and coffee morning this Saturday as part of the Somebodys Stranger campaign, in memory of Armagh man Gavin McNaney, who passed away two years ago.

It will take place from 10am until 2pm and it is a painless process which could potentially save a life.

Former St Catherines College teacher Gavin was just 37 years of age when he passed on November 18, 2017.

He had been diagnosed with Acute Lymphoblastic Leukaemia whilst teaching in Dubai.

Gavin spent months in hospital undergoing treatment and had a bone marrow transplant in London.

But after contracting a common cold and an infection to his lungs, his life was sadly cut short and he passed away peacefully with mum and dad, Nuala and Pat, by his side.

Friend Karl McQuaid has been raising funds and awareness after the passing of his life-long friend, whom he had first met when they both attended St Patricks Grammar School in Armagh.

He has been running registration events as part of his Somebodys Stranger campaign for nine months in Gavins memory and is keen to advise people just how easy it is to register .

He who would like to thank Leanne Armstrong and her colleagues at Translink for inviting them to come along told Armagh I : Joining the stem cell register is quick, easy and pain-free.

Potential donors have a swab taken of the inside of their cheeks with the whole process taking just a few minutes. They will then be added to DKMSs worldwide database and could be contacted at any time should they be a genetic match for a blood cancer sufferer anywhere in the world.

Those lucky enough to be a match would then be asked to donate their stem cells in a pain-free procedure similar to giving blood. This could save the life of the cancer sufferer.

Donations are at your own discretion at the event with all proceeds going to Leukaemia & Lymphoma NI Northern Irelands only charity dedicated to fighting blood cancers.

Those willing to join the register should be in general good health and aged between 18 and 55.

All are urged to come along on Saturday morning, when the city will be full of revellers for the annual Georgian Day event. Please take time to come along to the bus station and help make a huge difference.

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Translink in Armagh holding stem cell registration event in memory of Gavin McNaney - Armagh i

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(MENAFN - The Conversation) The global AIDS response has made significant progress in reducing HIV infections and AIDS-related deaths. New HIV infections dropped by16%from 1.9 million 2010 to 1.6 million in 2017. And the number of AIDS-related deaths decreased from 1.4 million to 940 000 in the same period.

But HIV/AIDS has not been brought under control and new infections continue to drive the epidemic. AIDS remains a leadingcause of deathin Africa.

Even if new infections are prevented,36.9 millionpeople with HIV around the world must take antiretroviral treatment to live a healthy life. While treatment is now as simple as taking a single pill a day, there are still many challenges to daily adherence, including ongoing stigma.

An ultimate solution would be a workable cure. At the recent Conference on Retroviruses and Opportunistic Infections researchersconfirmedthe second ever case of HIV remission or 'cure'. Known as the 'London patient', the person went into remission after a stem cell transplant as part of his treatment for cancer. He emerged from the procedure free of both his life-threatening Lymphoma and need for anti-HIV therapy.

The'Berlin patient' , Timothy Brown, made global headlines in 2008 when scientists announced that he had been cured of HIV. It's been 12 years since Brown was cured, after undergoing chemotherapy, total body irradiation and two stem cell transplants. Brown has been off treatment since the transplant and, after multiple tissue sampling procedures, has no remaining evidence of HIV reservoirs. The London patient is now the longest adult HIV remission after stem cell transplantation since the 'Berlin patient'.

This development is a triumph for medical science as well as for the London patient. But, as exciting as it is, stem cell transplant is a gruelling and dangerous procedure and isn't the magic bullet that will end HIV/AIDS. This is because it's unfortunately not a scalable, feasible cure for the 39 million people currently living with HIV.

The 'London patient' was HIV positive, but it was his Hodgkin's lymphoma that led to the need for a stem cell transplant.

The HI virus must link to a human host T cell in the blood or lymph nodes to replicate and infect the body. The virus attaches itself to a set of special links on the human T cell. If one of those links isn't available due to genetic mutations, the virus may find it harder to get an infection foothold.

One such genetic mutation occurs in a link called the 'CCR5 receptor'. Some people have this mutation naturally. The 'London patient', while on antiretroviral therapy and virally suppressed, had a bone marrow transplant as part of his lymphoma treatment. The bone marrow donor had the genetic mutation and passed it on to the 'London patient' through the procedure, making it more difficult for HIV to replicate.

The 'London patient' stopped taking antiretroviral therapy 16 months after the transplant. And 18 months later the virus remains undetectable. Usually, when a person with HIV stops treatment, the virus rebounds within the first month.

The achievement of remission in a second patient has provided further critical information to inform our understanding of how HIV infection occurs and the interaction between human cells and the virus.

As important as this work is, there's no scalable cure yet and it's also vital that researchers and countries keep putting effort into prevention. Important work continues to be done in this area.

As HIV cure research goes on, so does research into HIV prevention tools, such asPre-exposure prophylaxis(a daily pill that protects you from HIV infection) and the development of apreventative vaccine .

Two late stage vaccinetrialsare underway in sub-Saharan Africa. Results will be available in 2022. A preventative vaccine would also greatly enhance efforts to being the HIV epidemic under control.

A working cure, together with a preventative vaccine would be the ingredients for HIV eradication. Until then we need to get effective, accessible treatment for all who need it, while deploying the many prevention tools at our disposal.

MENAFN2811201901990000ID1099339725

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Why ending HIV still rests on a working cure -- as well as prevention - MENAFN.COM

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Researchers have designed a limb girdle muscular dystrophy 2A gene therapy preventing cardiotoxicity of calpain3 gene transfer.

Limb-girdle muscular dystrophy type 2A (LGMD2A or LGMDR1) is a neuromuscular disorder hat causes progressive muscle weakness and has no cure. The condition is caused by mutations in the calpain 3 gene (CAPN3). Previous experiments using adeno-associated viral (AAV) vectormediated calpain 3 gene transfer in mice indicated cardiac toxicity associated with the ectopic expression of the calpain 3 transgene. Now a team at French INSERM (Paris) and Karolinska Institute (Stockholm, Sweden) have created an improved gene therapy that can treat a form of muscular dystrophy in animal models without causing heart damage.

Calpain 3 is present in skeletal muscle and to a lesser extent in the heart. William Lostal and colleagues previously created a gene therapy for LGMD2A, but the treatment was toxic to the heart in mice because it activated calpain 3 in both the heart and skeletal muscles. In the current study, published in Science Translational Medicine, the researchers refined their approach by combining an adeno-associated viral vector expressingCAPN3with a heart-specific microRNA, which avoids heart toxicity while still correcting LGMD2A in the muscle.

Lostalet al.administered their therapy to a mouse model of muscle breakdown and calpain 3 and dysferlin deficiency and saw that it slowed the breakdown of skeletal muscle and restored the expression of calpain 3. The gene therapy was also well-tolerated when given to healthy macaques and boosted the expression of calpain 3 without causing heart toxicity. Finally, they found that titin a binding partner of calpain 3 showed differences in its calpain 3 binding sites across mice, macaques and humans, an observation that could explain the previously-observed heart toxicity. The interest for LGMD2A gene therapies in the market is high.

In May, Sarepta Therapeutics acquired a preclinical LGMD2A gene therapy programme developed by the Research Institute at Nationwide Childrens Hospital in Ohio. The calpain-3 programme uses a rhesus monkey-derived adeno-associated virus (AAVrh74) to directly deliver functional copies of the calpain-3 gene to patients skeletal muscle, intending to prevent further muscle damage via calpain-3 waste proteins.

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French-Swedish research team improves safety of DMD gene therapy - European Biotechnology

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MIT Provost Martin A. Schmidt said sharing the risk among several institutions will not only make possible work that would be difficult for a single institution to tackle, it will also encourage collaboration that accelerates the process of moving discoveries from lab to patient.

MIT researchers are developing innovative approaches to cell and gene therapy, designing new concepts for such biopharmaceutical medicines as well as new processes to manufacture these products and qualify them for clinical use, Schmidt said. A shared facility to de-risk this innovation, including production, will facilitate even stronger collaborations among local universities, hospitals, and companies and ultimately, such a facility can help speed impact and access for patients. MIT appreciates Harvards lead in convening exploration of this opportunity for the Commonwealth.

Richard McCullough, Harvards vice provost for research and professor of materials science and engineering, who also helped lead the project, said although the centers activity will revolve around science and manufacturing, its true focus will be on patients.

The centers overarching goal will be improving patient care, McCullough said. This would occur both by speeding access to the essential, modified cells that patients in clinical trials await, and by fostering discoveries through collaborations within the centers innovation space. The aim is that discoveries result in whole new treatments or improved application of existing treatments to provide relief to a wider universe of patients.

Organized as a private nonprofit, the center will be supported by more than $50 million pledged by its partners. It will be staffed by a team of at least 40, experienced in the latest cell-manufacturing techniques and trained in the use of the latest equipment. Among its goals is disseminating badly needed skills into the Boston life-sciences workforce.

We have to be sure that we are constantly feeding the industry with talented people who know the right things, so personally, I am very excited about education programs, Ligner said. Initiatives like [this center] are essential to advancing the industry because they help organizations build on one anothers advances. For example, the full potential of cell and gene therapies will only be realized if we collaborate to address challenges, such as manufacturing, improving access, accelerating innovation, tackling cost issues, and then sharing our learnings.

The new center emerged from conversations with state officials, including Gov. Charlie Baker and Attorney General Maura Healey, and industry sector leaders about ways to bolster Massachusetts preeminence in life science research and medical innovation. Those conversations sparked a two-year consultation process at the invitation of Garber and Harvard Corporation Senior Fellow Bill Lee, that was coordinated with state officials and included representatives from industry, academia, venture capital, area hospitals, and government.

Cell and gene therapies have the potential to revolutionize the global health system. Recently, in Sweden, the first patient received cell therapy outside of a clinical trial. Its the start of an incredible time in the industry and in human health.

Emmanuel Ligner, president and chief executive of GE Healthcare Life Sciences

Called the Massachusetts Life Sciences Strategies Group, members reached out to regional experts beginning in 2017to discover what fields they considered most important and how best to support them. Cell and gene therapy rose to the top because of the considerable excitement generated by activity already going on, its potential to help patients, and its high potential for future growth and innovation. Also important were the opportunities to spread the high cost of these technologies across multiple institutions and, while so doing, capture the collaborative power of housing each player in the development chain within a single facility.

The centers board of directors will be comprised of Harvard, MIT, and industry partners Fujifilm, Alexandria Real Estate Equities, and GE Healthcare Life Sciences. Other members will include Harvard-affiliated teaching hospitals Massachusetts General Hospital, Brigham and Womens Hospital, Beth Israel Deaconess Medical Center, Boston Childrens Hospital, and the Dana-Farber Cancer Institute; as well as the Commonwealth of Massachusetts and life-sciences company MilliporeSigma.

When you look at the constellation of players coming together, you really have the best universities and the best teaching hospitals and the best corporate players all supporting it, McGuire said, which I think is a great opportunity.

The facility intends to provide researchers and emerging companies outside the consortium with access to excess material, though organizers said they expect it to be in high demand by center partners.

The centers boost to the areas cell and gene therapy endeavors comes early enough that it should help maintain leadership over places like California and China, which have made clear their interest in life-science research, McGuire said.

I think getting this early mover advantage is going to be huge [in] developing the technology and the know-how and, ultimately, the intellectual property around it, McGuire said.

For Sharpe, the ultimate payoff will come from using cancer immunotherapys checkpoint blockade and other cell and gene therapies to save and improve lives.

We are seeing long-term benefits in some patients whove received checkpoint blockade, Sharpe said. There are patients who are more than a decade out and are melanoma-free. I think that it really has transformed patient care, quality of life, and longevity. So Im optimistic that the more we learn, the more were going to be able to do to help patients.

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Partnership aims to accelerate cell and gene therapy - Harvard Gazette

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27 Nov 2019

Twenty years ago, researchers took fibroblasts from the skin of eight Alzheimers patients, engineered them to produce nerve-growth factor, and slid them into each volunteers basal forebrain. They hoped the neurotrophin would halt or slow the neurodegeneration that robbed them of their memories, indeed their lives. The gamble failed and since then, scientists have shown little zest for gene therapy in neurodegenerative disorders. That is changing. As evident at this years Society for Neuroscience conference, held October 1923 in Chicago, gene therapy is back. Buoyed by success in treating spinal muscular atrophy in infants, scientists are flush with new ideasand funding.

What was once considered risky, expensive, and unlikely to succeed is now seen by many as risky, expensiveand quite likely to succeed. A growing number of scientists think gene-based therapies may have the best chance of slowing, or even preventing, neurodegeneration, especially for disorders caused by mutations in a single gene. SfN hosted a press briefing on gene therapy, plus many projects are active throughout the field beyond those showcased at the conference. There was no breaking clinical trial news at the annual meeting, but the scope and challenges of such therapies were outlined at the briefing moderated by Rush University s Jeff Kordower, Chicago, as well as a translational roundtable moderated by Asa Abeliovich, Columbia University, New York. Abeliovich recently co-founded Prevail Therapeutics, New York.

Going viral. Researchers are tweaking the capsid of adeno-associated viruses to optimize gene therapies for a multitude of disease. Shown here, AAV2.

From Zolgensma to Alzheimers? If the failure of the nerve growth factor therapy tempered enthusiasm for gene therapy (Mar 2018 news), then the success of AVXS-101, aka Zolgensma, reignited it. Developed by scientists at Nationwide Childrens Hospital, Columbus, Ohio, and AveXis, Bannockburn, Illinois, AVXS-101 uses an adeno-associated virus to deliver billions of copies of the survival motor neuron 1 gene to the brain. A small pilot trial tested the therapy in babies with spinal muscular atrophy (SMA) Type 1, the severest form of this neurodevelopmental disease. Lacking functional SMN1, these infants face progressive muscle weakness. Most die before their second birthday; those who live need a ventilator to breathe.

In Phase 1, AVXS-101 dramatically improved motor function of 15 treated infants; all were living 20 months later when historical data predicted only one would survive. Twelve babies who received the highest dose grew stronger within months, most sitting independently and rolling over. They hit the highest score on a scale of motor function, whereas untreated babies deteriorated. By 20 months, two of the treated babies had begun to walk (Mendell et al., 2017). The Food and Drug Administration approved zolgensma in May 2019. At SfN in Chicago, Petra Kaufmann, AveXis, played videos of the first patients treated with AVXS-101. Some four years later, they are walking, running, and appear to be playing almost normally. A video of a little girl walking downstairs with nary a hint of having SMA Type I visibly moved the audience.

Scientists say its a game-changer. It is really the tremendous success with SMA that has renewed interest in gene therapy, said Clive Svendsen, Cedars-Sinai Regenerative Medicine Institute, Los Angeles. Speaking with Alzforum before SfN, Bart De Strooper, Dementia Research Institute, London, said the same. The success in SMA patients of both gene therapy and antisense therapy has revived interest in the whole area, De Strooper said. Nowadays, researchers tend to lump gene therapy and antisense therapy under one moniker, i.e., gene-based therapy. The SMA antisense therapy nusinersen also works in babies with SMA Type 1 and is FDA-approved (Nov 2016 news; May 2018 conference news). Unlike gene therapy, antisense therapy needs to be delivered indefinitely.

How About Neurodegenerative Disease?At SfN, scientists outlined strategies for treating adults who face years of decline due to Alzheimers, amyotrophic lateral sclerosis, frontotemporal dementia, Huntingtons (HD) and Parkinsons diseases (PD), or other synucleinopathies. Some are being tested in clinical trials, others are in preclinical development. Some target specific losses or gains of function, others aim to rescue dying neurons more broadly. Scientists also believe that working on rare childhood diseases of lysosomal storage may give them an opening to treat this common phenotype in age-related neurodegeneration, as well.

Just this October, an ApoE gene therapy trial started enrolling. Led by Ronald Crystal at Weill Cornell Medical College, New York, it will inject adeno-associated virus carrying the gene for ApoE2 into patients with early to late-stage AD who inherited two copies of ApoE4. The idea is to flood their brains with the protective allele of this apolipoprotein to try to counteract the effects of the risk allele. AAV-rh10-APOE2 will be injected directly into the subarachnoid cisternae of participants brains. The Phase 1 trialwill recruit 15 patients with biomarker-confirmed AD. Beverly Davidson, Childrens Hospital of Philadelphia, has a similar ApoE2 gene therapy in preclinical development.

At SfN, Abeliovich detailed Prevails programs for forms of PD and for frontotemporal dementias that are caused by risk alleles. A trial has begun for a glucocerebrosidase-based gene therapy. The enzyme GCase is essential for lysosomes to function properly. People who have loss-of-function mutations in both copies of the GBA1 gene develop Gauchers, a lysosomal storage disease. The severest form starts in babies, most of whom die before age 2. Milder forms cause later-onset Gauchers, while heterozygous mutations in GBA1 increase risk for Parkinsons, making restoration of GCase an obvious strategy for PD. Some researchers are trying to develop ways to boost activity of the mutated enzyme (e.g., Oct 2019 news), whereas Abeliovich and colleagues have constructed AAV-9 vectors to deliver normal GBA1 into the brain to restore GCase production.

In preclinical studies, the AAV9-GBA1 construct PR001 rescued both lysosomal and brain function in models of GCase deficiency and of Parkinsons, Abeliovich said. In mice fed the GCase inhibitor conduritol epoxide (CBE), PR001 injected into the brain ventricles beefed up GCase activity and reduced glycolipid accumulation, which is a sign that lysosomes are functional. A single dose worked for at least six months. Similar results were seen in a commonly used model of Gauchers that expresses the V394L GBA mutation and only weakly expresses prosaposin and saposins, lysosomal proteins that metabolize lipids. In these 4L/PS-NA mice, PR001 made increased levels of active GCase, fewer lipids accumulated, and the mice were more mobile on a balance beam. 4L/PS-NA mice also accumulate -synuclein, the major component of Lewy bodies in PD and other synucleinopathies. In these mice, and also in A53T -synuclein mice made worse with CBE, PR001 halved the amount of insoluble -synuclein, Abeliovich reported at SfN.

In search of the right dose for humans, the scientists next turned to nonhuman primates. They injected PR001 into the cisterna magna in hopes AAV9 would broadly distribute throughout the brain. At the highest dose, 8 x 1010 capsids per gram of brain weight, exposure in the brain was similar to that seen in the mice. The virus permeated the spinal cord, frontal cortex, hippocampus, midbrain, and putamen.

Also in October, Prevail scientists began recruiting for a Phase 1/2 double-blind, sham-controlled trial to test this gene therapy in 16 people with moderate to severe PD, who have mutations in one or both copies of their GBA1 genes. Six patients each will receive a low or high dose of PR001A. Blood and CSF biomarkers to be analyzed at three and 12 months, and at follow-up, include GCase, lipids, -synuclein, and neurofilament light chain. Participants will also undergo cognitive, executive, and motor-function tests and brain imaging. A Phase 1/2 trial of PR001 in neuronopathic Gauchers, which affects the brain and spinal cord, will start soon, Abeliovich said.

Other groups are boosting dopamine production in Parkinsons by way of gene therapy. VY-AADC,developed by Voyager Therapeutics, Cambridge, Massachusetts, packages the gene for L-amino acid decarboxylase (AADC), which converts L-dopa into dopamine, in an AAV-2 vector that is delivered into the brain. Two Phase 1 open-label trials are testing safety and efficacy. Both the PD-1101 and PD-1102 trials use MRI to guide injections of the vector bilaterally into the putamina of 15 or 16 patients, respectively. According to preliminary results presented at the annual meeting of the American Academy of Neurology this past May, the virus penetrated half of the putamen and AADC activity, as judged by 18F-DOPA PET, increased by 85 percent in the latter study. Seven of eight treated patients reported improvement after a year, along with longer on time on L-DOPA, and shorter off time. Off time is the period when L-DOPA effects wear off and patients experience loss of motor control. RESTORE-1, a Phase 2 study of 42 patients, started in 2018 and will run to the end of 2020.

Long-Lived Gene Therapy. When a Parkinsons disease patient died eight years after neurturin gene therapy, the trophin was still being expressed in their putamen (top left) and substantia nigra (bottom left), where it corresponded with tyrosine hydroxylase activity (right). [Courtesy of Jeff Kordower.]

Also in PD, Kordower and colleagues plan to re-evaluate neurturin-based gene therapy. Previously, the gene for this neurotrophin was delivered in an AAV2 vector into the brains of Parkinson patients in Phase 1 and 2 trials. This did not improve motor function. Even so, in Chicago Kordower showed that in two patients who died eight and 10 years later, the inserted gene was still expressing neurturin and that dopamine levels were higher on the injected than the contralateral side of the substantia nigra/putamen. This shows us that long-term gene expression can be achieved in the human brain, said Kordower (see image above). He believes that by focusing delivery with ultrasound, or tweaking the capsid itself, he may be able to generate enough gene expression to improve function.

Separately, AAV-GAD, a gene therapy for PD that showed promise in Phase 2 (Mar 2011 news) was acquired by MeiraGTx, New York, which will continue to develop it in the U.S. and Europe, according to founder Samuel Waksal (Nov 2018 news).

For its part, Prevail has a gene transfer construct for frontotemporal dementia in the pipeline, as well. Called PR006, it carries GRN, the gene encoding progranulin, on an AAV9 vector. GRN mutations cause familial FTD and, much like GBA mutations, do their dirty work via lysosomal dysfunction. In Chicago, Abeliovich reported that PR006 boosted progranulin release from neurons derived from FTD-GRN patients, nearly doubling their levels of mature Cathepsin D, the lysosomal protease that chops progranulin into granulins and indicates healthy lysosomes. In progranulin knockout mice, PR006 restored brain GRN expression and progranulin secretion into the CSF. Abeliovich said he expects a Phase 1/2 clinical trial in FTD patients to start in early 2020.

The biotech company Passage Bio, Philadelphia, is planning for clinical trials early next year with its AAV-GRN vector. MeiraGTx, New York, is banking on a different approach for FTD. They have developed an AAV carrying UPF1, which encodes regulator of nonsense transcripts 1. This protein helps clear out aberrant RNAs through a process call nonsense-mediated decay. MeiraGTx hopes this will restore homeostasis to RNA processing. AAV-UPF1 will be trialed for FTD and all forms of ALS bar those caused by mutations in SOD1. For SOD ALS, Novartis, Basel, Switzerland, and REGENXBIO, Rockville, Maryland, have a vector in preclinical testing.

For his part, Svendsen is taking a different approach. His lab tackles ALS with ex vivo gene therapy. The idea is to engineer clinical-grade human stem cells to produce glial-derived growth factor, and inject them into the spinal cord, much like the early NGF studies did in AD. Svendsen hopes the cells will churn out enough of the neurotrophin to protect spinal cord motor neurons. In a Phase 1/2a trial, 18 ALS patients have received these cells into one side of their spinal cords, such that each person serves as his or her own control. If this works, they would regain mobility only on the injected side. The trial finished in October; Svendsen expects results to come out in a few months. In a follow-up study, the scientists are trying to do the same with induced pluripotent stem cells. This would allow them to transplant autologous cells into patients, avoiding immune rejection

Other groups are deploying gene therapy as a way to improve immunotherapy, shield neurons from stress, or even generate neurons from astrocytes to make up for those lost to neurodegeneration.Tom Fagan

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A glance around the walls of Barry J. Byrnes office reveals a lot about the pediatric cardiologist who runs thePowell Gene Therapy Center at University of Florida (UF).

In one corner is an unusual painting by 9-year-old Will Barkowsky of Jacksonville, Fla. Will, the first boy with Duchenne muscular dystrophy to takeSarepta Therapeutics exon-skipping medication Exondys 51 (eteplirsen), put together his oil-on-canvas masterpiece using the tire tracks of his wheelchair, making sure the colors didnt mix.

Nearby is a movie poster for The Ataxian an award-winning 2015 documentary by Kevin Schlanser and Zack Bennett about 17-year-old Kyle Bryant, who despite having Friedreichs ataxia embarks on a cross-country bicycle trip with three buddies.

Another movie poster advertises Extraordinary Measures, the 2010 tearjerker starring Brendan Fraser as John Crowley the father of two kids with Pompe disease and later, the founder of Amicus Therapeutics and Harrison Ford as fictional researcher Robert Stonehill, who discovers a treatment for the genetic disorder that eventually saves the lives of Crowleys children.

Theres also a model of a Blalock-Taussig shunt frequently used in congenital heart surgery, as well as one of an adeno-associated virus (AAV) vector, along with a prominent photo of Byrne with Ron Bartek, co-founder and director of the Friedreichs Ataxia Research Alliance (FARA).

Friedreichs ataxia is where were putting most of our efforts now, said Byrne, who spoke to BioNews Services publisher of this website at length during a recent visit to his lab in Gainesville, Fla.

Byrne, along with Jerry Mendell, MD, a neurologist with Nationwide Childrens Hospital in Columbus, Ohio, hosted a Nov. 20 webinar on gene therapy organized by the National Organization for Rare Disorders (NORD) and the American Society for Gene & Cell Therapy.

The two experts were introduced by Katie Kowalski, senior program manager for NORDs Educational Initiatives. The webinar, Understanding the Gene Therapy Process and Aftercare, was the fourth in a five-part series underwritten by Amicus and Sarepta, as well as two other companies, Avrobio and Bluebird Bio.

The final webinar in the series, Life After Gene Therapy, is scheduled for Dec. 18.

Mendell, who heads Nationwides Center for Gene Therapy, specializes in gene therapy research for Duchenne as well as limb-girdle muscular dystrophy, spinal muscular atrophy (SMA) and X-linked myotubular myopathy. He was a principal investigator for the Novartis therapy Zolgensma, which uses an AAV vector to carry a working version of SMN1, the mutated gene in people with SMA.

Zolgensma won approval from the U.S. Food and Drug Administration (FDA) in May 2019 as the first gene therapy to treat SMA in infants up to 2 years of age.

At $2.125 million per patient, the hour-long Zolgensma infusion is the most expensive medicine in history. The cost easily eclipses that of the only other FDA-approved treatment for SMA, BiogensSpinraza(nusinersen), which retails for $750,000 the first year and $375,000 every year after.

Many of my colleagues have been trying to make inroads for years, Mendell said. When we first got into the gene therapy domain, we were limited by technology. We could not make enough virus for the kind of impact were having now. But technology has improved, and we can now deliver genes through circulation to reach all muscles.

Regardless of the disease, he said, its extremely important to confirm the patients specific mutation before anything else.

This is critical, because you dont want to deliver the wrong kind of gene in a disease like Friedreichs ataxia. That goes for all gene therapy trials, he said. Next, we want to check for pre-existing antibodies, whether theyre acquired from the environment or from close contact. They bind to the AAV and block entry to the target organ.

Checking for those antibodies requires a blood test. It generally takes 4-7 days to return lab results a nailbiting time for patients and families, Mendell said, because theyre waiting to be approved for enrollment in the trial.

Byrne estimated that 50-60% of all individuals may have been exposed to AAV.

Prior exposure at any level to any AAV infection is an exclusion in most studies, he said, noting that people who travel frequently or who have respiratory or gastrointestinal conditions are particularly susceptible. We are learning a lot about what thresholds are effective. Its about 10% of newborns and about 50% of those of school age and adulthood.

Patients must also be in general good health except, of course, for the genetic disease being treated. MRI and blood tests are done to rule out diabetes or any evidence of heart, liver, or kidney problems.

We put the patient to sleep so theres really no pain involved, Mendell said. We also use local numbing medicine, even though the patient is asleep, so theres no pain or discomfort.

The Powell Gene Therapy Center was established in 1996 the year before Byrne joined UF by Nicholas Muzyczka, PhD, who performed groundbreaking work on AAVs in the 1980s. The center has a dozen individual labs working in neuroscience and molecular genetics.

Byrne said that because gene therapy fundamentally changes many of the bodys cells, screening is crucial.

This is often a one-way street, in that since the effects are long-lasting, other experimental studies may not accept patients who have received gene therapy of any kind in the past, Byrne said. One must have the clinical features required of the study and meet certain functional and age criteria.

To prepare for screening, patients or their parents must read the informed consent and understand what the risks and benefits are. Genetic counseling also may be required to determine whether a given mutation is amenable to gene therapy.

Baseline evaluations are done when its a muscular skeletal disease timed function tests as well as lab tests and a study schedule is established, he said. In many of our studies, we see the patients very frequently, almost every day for the first two weeks. They stay in the area for up to a month. Because were often dealing with rare populations, that makes it convenient for us to evaluate these patients.

Byrne noted that gene therapy is not necessarily durable for the lifespan of the patient. Because the delivered gene does not integrate into the cells own DNA, it is not passed down to newly formed cells.

Some cells, particularly in the liver and muscle, continue to grow throughout childhood and AAV doesnt integrate, so its progressively less effective unless the cells being targeted, as in SMA, are not dividing, he said. Thats an example where newborn screening is critically important to better outcomes.

Mendell said he generally starts patients on prednisone one day before receiving gene therapy in order to suppress liver inflammation, and keeps them on it for 60 days after.

When were in the room, the first thing that happens is the gene is delivered. You push a button and get started, he said. Obviously it must be the correct gene. Its in there, but you cant see it.

The actual gene is delivered by intravenous (IV) infusion with a pump over a 90-minute period, Mendell said; anything faster than that could potentially cause harmful side effects.

We put IVs in both arms for continuous delivery in case one side gets clogged up. We dont want anything to stop gene delivery, he said. Meanwhile, the patient is constantly monitored for vital signs. We invite the whole family to stay together, and thats reassuring. Theres anxiety about gene therapy, but the potential benefits generally outweigh any risks involved.

Some patients may develop nausea and vomiting in the first one-to-three weeks following treatment. For this reason, blood is taken every two weeks for three months to check for side effects.

Mendell said he knows patients are responding to gene therapy by doing testing. In the case of Duchenne, he uses the North Star Ambulatory Assessment, which includes 17 timed tests such as climbing stairs, rising from a sitting position, and walking or running 100 meters. In addition, neck control is a very good indicator of efficacy among Duchenne boys, he said.

The FDA anticipates that within the next 10 years, it will approve up to 40 gene therapies for rare conditions. Mendell said the benefits of gene therapy for one condition in particular, SMA, are undeniable.

This is an absolutely devastating disease. In type 1 SMA, patients usually dont survive past age 2, and about 50% are gone by age 1, he said. Initially there was concern about giving this to infants, but we told the FDA we needed to test infants in order to save lives.

Continuing results from Mendells pivotal Phase 1 trial (NCT02122952) in 15 type 1 infants and along-term extension study (NCT03421977) have changed the way people view gene therapys potential in general.

After four years, he said, every patient in our trial went from being unable to sit to being able to, and several are able to walk. One patient was treated 28 days after birth, and now four years later, hes off to school. What Barry and I do is very gratifying, and we thank our patients and their families for this opportunity.

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Ana holds a PhD in Immunology from the University of Lisbon and worked as a postdoctoral researcher at Instituto de Medicina Molecular (iMM) in Lisbon, Portugal. She graduated with a BSc in Genetics from the University of Newcastle and received a Masters in Biomolecular Archaeology from the University of Manchester, England. After leaving the lab to pursue a career in Science Communication, she served as the Director of Science Communication at iMM.

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Experts Barry Byrne, Jerry Mendell Lead NORD Webinar on Gene Therapy - SMA News Today

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DUBLIN, Nov. 25, 2019 /PRNewswire/ -- The "Gene Therapy - Market Analysis, Trends, and Forecasts" report has been added to ResearchAndMarkets.com's offering.

The Gene Therapy market worldwide is projected to grow by US$3.3 Billion, driven by a compounded growth of 32.7%.

Lentivirus, one of the segments analyzed and sized in this study, displays the potential to grow at over 25.3%. The shifting dynamics supporting this growth makes it critical for businesses in this space to keep abreast of the changing pulse of the market. Poised to reach over US$125.3 Million by the year 2025, Lentivirus will bring in healthy gains adding significant momentum to global growth.

Representing the developed world, the United States will maintain a 30% growth momentum. Within Europe, which continues to remain an important element in the world economy, Germany will add over US$133.3 Million to the region's size and clout in the next 5 to 6 years. Over US$117.2 Million worth of projected demand in the region will come from the rest of the European markets. In Japan, Lentivirus will reach a market size of US$6.5 Million by the close of the analysis period.

As the world's second largest economy and the new game changer in global markets, China exhibits the potential to grow at 39.2% over the next couple of years and add approximately US$797 Million in terms of addressable opportunity for the picking by aspiring businesses and their astute leaders.

Presented in visually rich graphics are these and many more need-to-know quantitative data important in ensuring quality of strategy decisions, be it entry into new markets or allocation of resources within a portfolio. Several macroeconomic factors and internal market forces will shape growth and development of demand patterns in emerging countries in Asia-Pacific, Latin America and the Middle East. All research viewpoints presented are based on validated engagements from influencers in the market, whose opinions supersede all other research methodologies.

Key Topics Covered:

1. Market Overview

2. Focus on Select Players

3. Market Trends & Drivers

4. Global Market Perspective

Competitors identified in this market include:

For more information about this report visit https://www.researchandmarkets.com/r/l8mwap

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

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Research and Markets Laura Wood, Senior Manager press@researchandmarkets.com

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(Reuters) - Eleven drugmakers led by Pfizer and Novartis have set aside a combined $2 billion to invest in gene therapy manufacturing since 2018, according to a Reuters analysis, in a drive to better control production of the worlds priciest medicines.

FILE PHOTO: A logo for Pfizer is displayed on a monitor on the floor at the New York Stock Exchange (NYSE) in New York, U.S., July 29, 2019. REUTERS/Brendan McDermid

The full scope of Novartis (NOVN.S) $500 million plan, revealed to Reuters in an interview with the companys gene therapy chief, has not been previously disclosed. It is second only to Pfizer (PFE.N), which has allocated $600 million to build its own gene therapy manufacturing plants, according to filings and interviews with industry executives.

Gene therapies aim to correct certain diseases by replacing the missing or mutated version of a gene found in a patients cells with healthy copies. With the potential to cure devastating illnesses in a single dose, drugmakers say they justify prices well above $1 million per patient.

But the treatments are also extremely complex to make, involving the cultivation of living material, and still pose a risk of serious side effects.

Drugmakers say building their own manufacturing plants is a response to rising costs and delays associated with relying on third-party contract manufacturers, which are also expanding to capitalize on demand.

They say owning their own facilities helps safeguard proprietary production methods and more effectively address any concerns raised by the U.S. Food and Drug Administration (FDA), which is keeping a close eye on manufacturing standards.

Theres so little capacity and capability at contract manufacturers for the novel gene therapy processes being developed by companies, said David Lennon, president of AveXis, Novartiss gene therapy division. We need internal manufacturing capabilities in the long term.

The approach is not without risks.

Bob Smith, senior vice president of Pfizers global gene therapy business, acknowledged drugmakers take a leap of faith when they make big capital investment outlays for treatments before they have been approved or, in some cases, even produced data demonstrating a benefit.

The rewards are potentially great, however.

Gene therapy is one of the hottest areas of drug research and, given the life-changing possibilities, the FDA is helping to speed treatments to market.

It has approved two so far, including Novartiss Zolgensma treatment for a rare muscular disorder priced at $2 million, and expects 40 new gene therapies to reach the U.S. market by 2022.

There are currently several hundred under development by around 30 drugmakers for conditions from hemophilia to Duchenne muscular dystrophy and sickle cell anemia. The proliferation of these treatments is pushing the limits of the industrys existing manufacturing capacity. Developers of gene therapies that need to outsource manufacturing face wait times of about 18 months to get a production slot, company executives told Reuters.

They are also charged fees to reserve space that run into millions of dollars, more than double the cost of a few years ago, according to gene therapy developer RegenxBio.

As a result, companies including bluebird bio (BLUE.O), PTC Therapeutics (PTCT.O) and Krystal Biotech (KRYS.O) are also investing in gene therapy manufacturing, according to a Reuters analysis of public filings and executive interviews.

They follow Biomarin Pharmaceutical Inc (BMRN.O), developer of a gene therapy for hemophilia, which constructed one of the industrys largest manufacturing facilities in 2017.

The FDA is keeping a close eye on standards.

This comes amid the agencys disclosure in August that it is investigating alleged data manipulation by former executives at Novartis AveXis unit.

AveXis had switched its method for measuring Zolgensmas potency in animal studies. When results using the new method didnt meet expectations, the executives allegedly altered the data to cover it up, the FDA and Novartis have said.

One of the former executives, Brian Kaspar, denied wrongdoing in a statement to Reuters. Another, his brother Allan Kaspar, could not be reached for comment.

Novartis and the FDA say human clinical trials, which found Zolgensma effective in treating the most severe form of spinal muscular atrophy in infants, were not affected. Novartis also says its investments in gene therapy production started long before it became aware of the data manipulation allegations.

But the scandal has highlighted the importance of having a consistent manufacturing process for gene therapies, industry executives say.

According to four of them, the FDA has stressed in recent meetings the need for continuity in production processes all the way from the development of a drug to its commercialization.

By bringing production in-house, drugmakers may avoid pitfalls such as the need to switch to a larger facility if contract manufacturers capacity proves limited, executives say.

The FDA is finalizing new guidelines for gene therapy manufacturing, expected at the end of the year.

Manufacturing consistency is always a major concern for the agency, FDA spokeswoman Stephanie Caccomo told Reuters.

Highlighting the pressures on the industry, Sarepta Therapeutics (SRPT.O), which largely outsources manufacturing, delayed a clinical trial of its Duchenne treatment in August, telling investors it wanted to avoid any questions from regulators about consistency in producing its therapy at commercial scale.

Between the trade secrets, the cost schedules and the time lag, it makes a whole lot of sense, if you can do it, to build out your own facilities and more and more gene therapy companies have started to do that, said Krish Krishnan, chief executive of Krystal Biotech Inc.

Krystal, which is developing therapies for rare skin diseases, has built one manufacturing facility and plans to invest more than $50 million in a new one it will start constructing in December.

MeiraGTx (MGTX.O), which focuses on gene therapies for eye conditions, estimates it is currently spending roughly $25 million a year on manufacturing, including process development.

Despite such moves, however, contract manufacturers like Lonza (LONN.S) and Thermo Fisher (TMO.N) are confident their businesses will continue to grow due to the strength of demand.

Thermo Fisher has told investors its Brammer gene therapy manufacturing division, acquired in May, could soon earn $500 million in revenue a year, double its projected 2019 earnings. Lonza CEO Marc Funk is also optimistic.

Demand in gene therapy has increased, he said in an interview. We believe this is going to continue in the coming years.

Reporting by Carl O'Donnell in New York and Tamara Mathias in Bengaluru; Editing by Tomasz Janowski, Michele Gershberg and Mark Potter

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