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 ...

Gene therapy is a medical treatment that works by changing or fixing a person's genes to treat or prevent disease ...

Gene therapy holds the promise of preventing and curing disease by manipulating gene expression within a patient's cells. However, to be effective, the new gene must make it into a cell's nucleus. The ...

Two dose-limiting toxicities have been recorded in uniQure’s trial evaluating an investigational AAV gene therapy designed to ...

Two-year follow-up data demonstrate sustained benefit, including improvements in pain, fatigue and bone health, following single low dose of ...

Data represents up to 8.5 years of follow-up and are consistent across age, dose, and genotypeBLA resubmitted to U.S. FDA in January 2026; ...

This sponsored article was provided by a partner and is not editorial content from Los Angeles Magazine or the Engine Vision Media Network. Every cell in your body holds a unique genetic code within ...

There are currently no approved disease-modifying therapies for MPS IIIA.

When Genenta Science listed on the Nasdaq at the tail end of 2021, the Milan-based biotech was squarely focused on its pipeline of cell-based gene therapies. | Four years after listing on the Nasdaq, ...

Duchenne muscular dystrophy (DMD) is a neuromuscular disorder that results from mutations in the DMD gene. Gene therapies for DMD change genetic material in a person’s body to treat this condition.