The approach is “very exciting,” but one expert says a lot more research is needed, especially around safety, before clinical ...

In a 15-patient, Phase 1, first-in-human trial, a one-time CRISPR-Cas9 gene-editing therapy safely reduced LDL cholesterol ...

From revolutionary CRISPR treatment to heavy drinking dangers, here's what you need to know about the latest health research.

Scientists at St. Jude Children's Research Hospital and Northwestern University identified a previously unknown treatment ...

A CRISPR-based gene therapy showed improvement in lipids in difficult-to-treat patients, but one study raised flags after a death last month in a trial of an unrelated gene therapy.

The Food and Drug Administration said Friday it will add a new warning and other limitations to a gene therapy for Duchenne’s ...

EU regulator says treatment could fill major gap for patients who lack a suitable stem cell donor and leads to sharp drops in ...

Children with hereditary deafness regained their hearing thanks to a type of gene therapy, a new study published on Wednesday found. In a clinical trial, co-led by investigators from Mass Eye and Ear, ...

While gene therapy for cystic fibrosis is still in the research phase, researchers aim to correct the defective gene responsible for the disease. Current research is promising. Cystic fibrosis (CF) is ...

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 ...