gene therapy
Bluebird Bio defends as ‘simple and human’ its pricing of costly gene therapy for beta thalassemia
[J]ust as we spent the past years cracking the code to make gene therapy work, we now need to work ...
Gene therapy may offer cure for debilitating ‘bubble boy disease’
Omarion was born with a rare genetic disorder called X-linked severe combined immunodeficiency (SCID), better known as the “bubble boy ...
Trio of gene therapies seeks to reverse age-related diseases to make us ‘healthy, youthful later in life’
The legendary synthetic biologist Dr. George Church and team at the Wyss Institute at Harvard University took a first step towards ...
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 ...
Podcast: CRISPR advances, HIV cures and a perfect predator for superbugs
Exploring the latest advances in medical biotechnology including CRISPR-based gene therapies, infection-fighting viruses and a cure for HIV. Plus reflections ...
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 ...
Animal safety concerns prompt FDA to halt study testing Zolgensma gene therapy for spinal muscular atrophy
The Food and Drug Administration has halted a clinical trial of Novartis’ Zolgensma gene therapy due to a safety concern ...
Viewpoint: Netflix’s 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 ...
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 ...
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 ...
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 ...
Answering key questions about CRISPR gene therapies
What is gene editing? Genome editing (also known as gene editing) is a group of technologies that provide scientists with ...
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 ...
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 ...
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 ...
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 ...
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 ...
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 ...
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 ...
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 ...
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 ...
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 ...
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 ...
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 ...
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 ...
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 ...
Viewpoint: It’s time for a debate on precision medicine and its failed promises
Although some niche applications have been found for precision medicine, and gene therapy is now becoming a reality for a ...
