3-31-2019 thals patient

Gene therapy for beta-thalassemia blood disorder one step closer to European approval

Adam Feuerstein | 
The first gene therapy to treat a rare blood disorder is one step closer to approval [March 29] following a ...
gene

Why gene therapy’s potential is attracting intense interest from Big Pharma

Andrew Bary | 
Big Pharma has been struggling for years to produce drugs that are major breakthroughs in medicine. For these companies, gene ...
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Viewpoint: Call for moratorium on heritable gene editing reminds us of gene therapy’s troubled early days

Greg Licholai | 
A group of influential scientists have urged that clinical use of CRISPR be put on hold in the wake of ...
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Inside the quest to use gene therapy to cure a form of congenital blindness

Jean Bennett, Katherine High | 
[I]nherited retinal disease affects thousands of people around the world—people who are born with poor vision and then slowly lose ...
alzheimers

Alzheimer’s disease targeted with gene therapy trial

Shelly Fan | 
There’s a test for Alzheimer’s risk that genetic counselors don’t like to talk about. It’s not that they’re hiding the ...
2-26-2019 alzheimers trial bathe brains genes

Gene therapy could offer ‘one-time genetic tune-up’ for Alzheimer’s patients

Antonio Regalado | 
No one knows for certain what causes Alzheimer’s disease. But one fact about the condition has gained nearly irrefutable status ...
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Gene from controversial CRISPR baby experiment could deliver new stroke treatment

Karen Weintraub | 
A widely criticized experiment last year saw a researcher in China delete a gene in twin girls at the embryonic ...
2-18-2019 jim wilson mini e x

Pioneering ‘refugee’ from gene therapy’s darker days ‘kept the faith’

Damian Garde | 
Gene therapy, now a pillar of biotechnology with the potential to cure deadly diseases, was once a cautionary tale of ...
2-19-2019 angelman clinic feature

Rare brain disorder—Angelman syndrome—could be treated with gene therapy in the womb

Ian Sample | 
Scientists are developing a radical form of gene therapy that could cure a devastating medical disorder by mending mutations in the brains ...
2-18-2019 british

First human test for gene therapy targeting most common cause of blindness

Fergus Walsh | 
A woman from Oxford has become the first person in the world to have gene therapy to try to halt ...
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First gene-editing experiments on adults suffering from rare metabolic disorders offer promising early results

Carolyn Wilke | 
In late 2017, scientists first began attempting to edit the genes of adults to treat rare genetic disorders. Preliminary results ...
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Chinese scientists aren’t keeping tabs on experimental gene therapy patients, report says

George Dvorsky | 
Gene therapies are very much at their preliminary stages of development, so it would make sense to keep tabs on ...
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Can gene therapy offer a cure for sickle-cell disease?

Carolyn Johnson | 
[I]n November, six months after [21-year-old Manny] Johnson became the first patient to receive an experimental therapy aimed at curing ...
IMG

Inside a couple’s quest to pay for an experimental gene therapy to save their children

Antonio Regalado | 
“We need your help, we really do,” Gary [Landsman] says, his voice breaking. The Landsmans’ two sons—Benny, then 18 months, ...
Gene therapy for autism? Rare Angelman syndrome could answer key questions

Gene therapy for autism? Rare Angelman syndrome could answer key questions

Benjamin Philpot, Matthew Judson | 
[T]he success of genetic therapies [for autism] is not guaranteed. Even if disrupted genes can be replaced, repaired or otherwise ...
SOG

CAR-T cell therapy and the promise of immune cells engineered to fight cancer

David Haas | 
The National Cancer Institute estimates that more than 1.7 million people will be diagnosed with cancer this year. To cope ...
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Can we afford gene therapy’s million-dollar price tags?

Sumathi Reddy | 
[4-year-old] Caspian was born with a rare, inherited eye disorder called Leber congenital amaurosis, which results in the progressive deterioration ...
nobel

Were ‘crucial contributors’ snubbed in awarding Nobel prize for cancer immunotherapy?

Sharon Begley | 
[I]t’s rare that Nobel announcements don’t produce grumblings about who was left out, and this year was no exception. At ...
FDA

Seeking a new path for FDA regulation of revolutionary medical treatments

Kate Bachelder Odell | 
Researchers are developing therapies that could permanently alter a patient’s genes... . The early results are promising. Patients would live ...
gene therapy

Bringing us closer to ‘once-in-a-lifetime’ hemophilia treatment with experimental gene therapies

Gina Kolata | 
Scientists are edging closer to defeating a longtime enemy of human health: hemophilia, the inability to form blood clots. After ...
gene therapy

Mainstream medicine? US health officials want to ease restrictions on gene therapy experiments

Marilynn Marchione | 
U.S. health officials are eliminating special regulations for gene therapy experiments, saying that what was once exotic science is quickly ...
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Examining the legacy of W. French Anderson—’father of gene therapy’ and convicted child molester

Sharon Begley | 
[Dr. W. French] Anderson has been hailed as the father of gene therapy and was honored at George H.W. Bush’s ...
dmd

Duchenne muscular dystrophy gene therapy trial put on hold

Kate Sheridan | 
Sarepta Therapeutics’ clinical trial for a gene therapy to treat Duchenne muscular dystrophy has been paused by the Food and ...
gene therapy womb

Are we close to gene therapy in the womb?

Angela Chen, Arthur Caplan | 
[Recently] scientists reported that they were able to treat a serious genetic disorder in the womb — in mice. It sounds like ...
PRinc photo of fetus at weeks

Deadly brain disease could be treated in the womb with gene therapy

Alexander Burik | 
A research team from the UK and Singapore showed that a neurodegenerative condition called Gaucher disease, which can be fatal, ...
enhance

Viewpoint: We’re ‘nowhere close’ to being ready to edit human genomes

Alireza Edraki | 
Genome meddling to cure diseases is often worth the risk, but nothing else is just yet ...
space travel

How gene therapy could help astronauts survive deep space deadly radiation

David Warmflash | 
Over the past five decades, space travel advocates have been pushing to expand our footprint in space. They dream about lunar ...
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