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

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

The Boston biotech founded by Harvard professor David Sinclair is testing whether cellular reprogramming can turn back the ...

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

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 ...

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

Researchers announce breakthrough results for AMT-130, the first disease-modifying treatment for Huntington's disease, with ...

DURHAM, N.C. (WTVD) -- Duke Eye Center has become the first academic medical center in the country to implant a new cell-based gene therapy for a rare eye disease called MacTel. This procedure offers ...

Oncologists' experience with CGT has increased, with more patients treated and earlier lines of therapy being utilized.

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

BioMarin no longer expects to hit its $4 billion revenue target by 2027, citing various market factors such as impending ...