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 ...

Dustin Vidrine's sight is disappearing — shrinking, really. He has retinitis pigmentosa, a rare, inherited eye disease that runs in his family. By the time he was in his 20s, he noticed that his ...

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 ...

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 ...

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, ...

The special properties of methylcellulose foam could make it a vehicle for bedside genetic engineering, according to a proof-of-principle study from bioengineers at Fred Hutch Cancer Center. In the ...

Enh3ance trial results indicate DTX301 reduces ammonia levels by 18% and supports dietary liberalization in patients with OTC deficiency.

NEW ORLEANS — New data from a first-in-human trial of a CRISPR-based gene therapy for cholesterol disorders are giving lipids experts reason for optimism about the approach for treating cardiovascular ...

Personalized therapies for diabetes, while showing promise in international studies, is met with healthy skepticism from ...

In addition to new medications to treat this enzyme deficiency, research is underway on easier hormone sampling and fixing the gene mutation that causes CAH.