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

In a world-first trial, scientists used a one-off CRISPR gene edit to switch off a liver “fat brake” gene, slashing stubborn ...

A single infusion of CRISPR Therapeutics' experimental gene therapy was safe and reduced levels of harmful LDL cholesterol ...

Researchers report early but encouraging results on an experimental gene-editing treatment for high cholesterol.

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.

Fifteen-year-old Jayden Wilsey of Falmouth is the first person at Boston Children's Hospital to receive human gene therapy for sickle cell disease, following approval of the therapy in 2024 by the U.S ...

An innovative method of gene therapy to treat hearing and balance disabilities caused by impaired function of the inner ear ...

In 1962, a pediatric neurology resident at Columbia's Neurological Institute and his colleagues in the College of Physicians and Surgeons published a detailed paper in Pediatrics describing a new ...

A naturally occurring gene called Cyclin A2 (CCNA2), which turns off after birth in humans, can actually make new, functioning heart cells and help the heart repair itself from injury, including a ...

In a Phase 1, first-in-human trial, a one-time infusion of an investigational CRISPR-Cas9 therapy targeting angiopoietin-like protein 3 (ANGPTL3) was safe and reduced LDL cholesterol by nearly 50% and ...