Gene therapies have the potential to cure some diseases, but they are extraordinarily expensive. Location can also be a big ...

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 ...

A pioneering gene therapy could help treat a rare seizure disorder called Dravet syndrome, according to new clinical trial ...

RGX-121 was rejected in early February, with Regenxbio listing the primary reasons given by the FDA as concerns about the ability to properly define a patient population, the use of a natural history ...

Since scientists first discovered that human immune cells could be modified to become cancer-fighting agents, they've been trying to engineer a cell that's effective against solid tumors, which ...

A single one-time gene therapy could free patients with α-thalassemia, a rare and debilitating blood disorder, from the burden of lifelong transfusions. A single one-time gene therapy could free ...

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 ...

In a 15-patient, Phase 1, first-in-human trial, a one-time CRISPR-Cas9 gene-editing therapy safely reduced LDL cholesterol and triglycerides in people with difficult-to-treat lipid disorders, ...

Proof-of-concept trial in a single patient shows that cells can survive transplantation without immunosuppression ...

Four decades have passed since cochlear implants gave infants born deaf the ability to hear. Now, gene therapy promises to restore natural hearing for those born with a rare form of deafness, and the ...