A 3-year-old girl who underwent a breakthrough gene therapy treatment to treat profound hearing loss can hear on her own, two ...
“Beyond UniQure, the pipeline includes highly promising approaches that will ideally move the goal from merely managing ...
A new study has brought scientists one step further in the direction of developing a cure for a brutal group of rare brain ...
A one-time gene therapy using a patient’s own stem cells has effectively cured a deadly immune disorder in 95% of treated ...
Long-term follow-up shows 95% success rate, no serious complications in largest ADA-SCID gene therapy study to date ...
Stevie Ringel is the founder of the Kizuna Foundation, an organization seeking a treatment for a genetic retinal mutation ...
Adeno-associated virus (AAV)–mediated gene therapy was one of the focal points of this year's Muscular Dystrophy Association conference. Here, Barry Byrne, MD, PhD, University of Florida, speaks to ...
News Medical on MSN
Gene therapy shows long-term success in children with rare immune disorder
Results from the largest cohort of children who received a gene therapy for a rare immunodeficiency condition have shown the ...
As newborn screening and rapid DNA sequencing become routine, we are poised to catch and treat inherited diseases at their earliest stages. Today, we can intervene in the first days or weeks of life.
Immusoft of CA, a clinical stage, engineered B cell company, today announced a historic achievement: the safe and well-tolerated re-dosing of a patient with a gene modified therapeutic product ...
New research found that gene therapy for children with "bubble boy disease," or SCID, was successful in 95% of trial ...
29d
Gene editing is being sold on the promise of healthier babies — but what are the ethical concerns?
The race to advance gene editing tech continues, spurred on by starry-eyed Silicon Valley investors, entrepreneurs and even ...
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