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

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 ...

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.

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

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 ...

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, ...

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 ...

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 the ...