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CAR-T therapy raises difficult question: ‘What is the price of a human life?’

A new type of therapy for certain blood cancers is ingenious but it raises important questions about its cost, both ...
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Patient’s ‘serious’ kidney injury halts gene therapy trial for Duchenne muscular dystrophy

The Food and Drug Administration has halted a clinical trial involving a Duchenne muscular dystrophy gene therapy from Solid Biosciences ...
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Rejuvenate Bio’s gene therapy shows promise at reversing aging—in mice

Harvard synthetic biology pioneer George Church generated some buzz last year when he co-founded Rejuvenate Bio with the goal of ...
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Viewpoint: Netflix’s new horror movie ‘Eli’ is a fright. But why did they have to ‘tarnish gene therapy’?

Attempts to treat genetic disease shouldn’t be the stuff of horror films ...
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Gene therapy pioneer James Wilson makes a comeback after disastrous start

Twenty years ago, a young man named Jesse Gelsinger was injected with a large dose of gene-shuttling viruses designed in ...
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Gene editing used in China in a failed but promising attempt to cure HIV

Scientists in China have used CRISPR gene-editing technology to treat a patient with HIV, but it didn't cure the patient, ...
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A businessman’s daughter has an ‘ultra-rare’ disease, so he immersed himself in science to find a gene therapy cure

Simon and Nina Frost had spared no expense, taking Annabel to all the best neurologists around the country. Finally a ...
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We can identify ‘bad’ genes. Why can’t we use CRISPR gene editing to get rid of them?

Gene therapy 2.0 using CRISPR to knock out harmful genes sounds is a great idea--if and when it works. But ...
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Powerful CAR-T duo uses artificial ‘claws’ to grab and destroy HIV

Lengtigen, a biotech company based in Gaithersburg, MD, teamed up with researchers at the Albert Einstein College of Medicine in ...
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Application for pricey gene therapy Zolgensma contained manipulated data, but drug will remain on market, FDA says

The Food and Drug Administration said Tuesday [August 7]  that Novartis submitted manipulated data as part of its application for ...
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5 ways we could pay for expensive life-saving gene therapies

We have arrived at a special moment in health care. Innovative, life-changing gene therapies are here that will cure or ...
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$2.1 million gene therapy is most expensive drug in history

The federal Food and Drug Administration has approved a gene therapy for a rare childhood disorder that is now the ...
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How this promising gene therapy for a rare neuromuscular disease was fueled by passionate parents and a dog

The gene therapy will be too late for Joshua Frase. But it will be his legacy ...
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Why we shouldn’t view gene therapy as a treatment of last resort

Providers and payers must ask themselves a two-pronged question about gene therapy: Who should be treated and when? … The ...
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Gene therapy’s fight against ‘bubble boy’ disease may have yielded a safe cure

“Cure” is a strong word, but the authors are confident that it has been achieved ...
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Gene therapy could be revolutionary—if we can figure out how to pay for it

Dozens of revolutionary gene therapies that mend faulty strands of DNA are on their way, bringing the power to eliminate ...
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Can CAR-T cell therapy tackle solid tumors too?

Last summer, [Cecelia] Barron’s cancer went from stage 2 to stage 3 oligodendroglioma. Behnam Badie, her surgeon at City of Hope, ...
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Gene therapy for beta-thalassemia blood disorder one step closer to European approval

The first gene therapy to treat a rare blood disorder is one step closer to approval [March 29] following a ...
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Why gene therapy’s potential is attracting intense interest from Big Pharma

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

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

[I]nherited retinal disease affects thousands of people around the world—people who are born with poor vision and then slowly lose ...
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Alzheimer’s disease targeted with gene therapy trial

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

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

A widely criticized experiment last year saw a researcher in China delete a gene in twin girls at the embryonic ...
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Pioneering ‘refugee’ from gene therapy’s darker days ‘kept the faith’

Gene therapy, now a pillar of biotechnology with the potential to cure deadly diseases, was once a cautionary tale of ...
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Rare brain disorder—Angelman syndrome—could be treated with gene therapy in the womb

Scientists are developing a radical form of gene therapy that could cure a devastating medical disorder by mending mutations in the brains ...
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First human test for gene therapy targeting most common cause of blindness

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

In late 2017, scientists first began attempting to edit the genes of adults to treat rare genetic disorders. Preliminary results ...