Three 2026 Breakthrough Prize winners reflect on developing Luxturna, a gene therapy that treats blindness caused by rare ...
Researchers in the lab of Hans-Peter Kiem, MD, PhD, at Fred Hutch Cancer Center have devised a method that could one day treat genetic hematologic disorders by correcting how the body makes blood ...
Researchers have found a promising new method for gene therapy. They successfully restarted inactive genes by bringing them closer to genetic switches on the DNA called enhancers. The intermediate ...
The FDA has approved Otarmeni, the first-ever gene therapy for genetic hearing loss caused by the OTOF gene, for both ...
The search for gene therapies to treat rheumatoid arthritis (RA) has moved in fits and starts over the past two decades, with trials starting and stopping, and their focus shifting to osteoarthritis ...
Vence L. Bonham Jr., Acting Deputy Director at the National Human Genome Research Institute (NHGRI) and an investigator in the National Human Genome Research Institute’s (NHGRI) Division of Intramural ...
The FDA has approved Otarmeni, the first gene therapy for genetic hearing loss, through its National Priority Voucher program ...
Four patients with infantile-onset Pompe’s disease received a single intravenous injection of an adeno-associated virus serotype 9 vector carrying codon-optimized complementary DNA encoding human acid ...
As we continue our series exploring genetic medicine, both in this story and in Destiny's Child No Longer: Rewriting Genetic Fate, gene therapy stands at a crossroads. On June 16, Sarepta Therapeutics ...
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