The small study in patients with a rare disorder that causes liver and lung damage showed the potential for precisely ...

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 ...

Luxturna, a gene therapy for a rare disorder that causes ... But pharmaceutical companies have learned to use these techniques to their advantage. Novartis brought together experts from academia ...

NY resident Sebastien Beauzile was treated with a pioneering new gene therapy approach called Lyfgenia, curing his sickle ...

In a preclinical study, researchers at Children's Hospital of Philadelphia (CHOP) demonstrated a novel gene therapy with ...

Researchers at Kumamoto University have successfully executed a practical trial of a new genome-editing technique that allows ...

UNC Charlotte researchers are contributing to pioneering breakthroughs in cancer therapeutics, communicable disease ...

Several inherited disorders cause those cells to form incorrectly, leading to vision impairments of varying severity, but new gene therapy techniques could reverse those conditions. "Treatments ...

Several inherited disorders cause those cells to form incorrectly, leading to vision impairments of varying severity, but new gene therapy techniques could reverse those conditions. Treatments for ...

Regeneron’s investigational gene therapy has been tied to notable hearing improvements in 10 of 11 children who were treated for a rare genetic condition that causes hearing loss. “You can see ...

This latest gene therapy offering is Casgevy ... Also, the low-MRI technology serves as an alternative to radiation imaging techniques such as X-rays that pose a higher risk to children.