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 ...

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

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 ...

Insmed has launched a first-in-human Phase 1 trial testing its experimental ALS gene therapy INS1202 in people with the disease.

Diseases that affect the retina, the light-sensitive layer at the back of the eye, are a significant cause of visual impairment and blindness. Gene therapy holds promise for treating some of these ...

Ultragenyx said that the trial of the gene therapy will continue while data from the second primary endpoint is collected.

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 ...

SAR446268 is a one-time adeno-associated viral gene therapy that delivers a vectorized RNA interference to silence DMPK expression. The Food and Drug Administration (FDA) has granted Fast Track ...

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