Gene Therapy
Gene therapy offers hope for untreatable CTE brain disorder
[Researchers from Weill Cornell Medical College] published a new study (“Anti-Phospho-Tau Gene Therapy for Chronic Traumatic Encephalopathy”) in Human Gene Therapy that ...
Virus used to deliver gene therapy treatments may increase liver cancer risk, study suggests
Just as gene therapy finally seems to be living up to its promise, a study has revived a lingering worry ...
Forecasting: 2020 could bring major advances in gene therapies and pig-to-human organ transplants
Here are two stories in biotech we’re keeping our eyes on. Although successes likely won’t come to fruition this year ...
Will 2020 see the debut of promising gene therapy for hemophilia A? It’s up to the FDA.
Hemophilia A gene therapy has been twenty years in the making ...
2019 offered ‘eclectic’ mix of potentially breakthrough treatments for genetic diseases, from cystic fibrosis to sickle cell
2019 was a very good year for new treatments of genetic diseases ...
Would price controls hamper research on gene therapy and other innovative treatments?
Over the next decade, it is a near certainty that we will have gene-therapy cures for deadly inherited disorders such ...
$1 million for anti-aging gene therapy? ‘Dubious’ treatment raises ethical concerns
Would you pay $1 million and fly to South America for a chance to live longer? Libella Gene Therapeutics, a ...
Viewpoint: Spinal muscular atrophy treatment Zolgensma a gene therapy trailblazer, despite controversy
In August, the US Food and Drug Administration (FDA) gave drug manufacturer AveXis, Inc, a subsidiary of Novartis AG, a ...
Viewpoint: Experimental gene therapy saved my sons’ eyesight
Not long after we brought my newborn son, Anthony, home from the hospital, we noticed his eyes kept darting to ...
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 ...
Expensive 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 ...
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 ...
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 ...
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 ...
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, ...
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 ...
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 ...
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 ...
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, ...