In honor of World Blindness Awareness Month, "GMA" is spotlighting a breakthrough gene therapy that's helping some blind ...

In a world first, a bespoke gene-editing therapy benefitted one child. Now reseachers plan to launch a clinical trial of the ...

In 1962, a pediatric neurology resident at Columbia's Neurological Institute and his colleagues in the College of Physicians ...

A 3-year-old girl who underwent a breakthrough gene therapy treatment to treat profound hearing loss can hear on her own, two ...

In a first for adults in Singapore, scientists are conducting gene editing trials on heart patients to correct defects at ...

The U.S. Food and Drug Administration has placed a clinical hold on Intellia Therapeutics' two late-stage trials testing an ...

UniQure's therapy, called AMT-130, reduced disease progression by 75% at 36 months in patients who received a high dose.

The FDA has officially placed two of Intellia’s Phase III studies under clinical hold after the biotech reported earlier this ...

Duke Eye Center has become the first academic medical center in the country to implant a new cell-based gene therapy for a ...

Up to 60% of congenital and early-onset hearing loss is caused by genetic mutations in an inherited gene, and gene therapy ...

Intellia earlier this year reported a similar grade 4 liver enzyme elevation associated with the gene therapy nexiguran ...

Some of the most expensive drugs currently in use are gene therapies to treat specific diseases, and their high cost limits ...