We have written about the promise of gene therapy to correct genetic disease many times. An exciting development happened ...

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

The FDA is said to be making a U-turn on uniQure’s one-time gene therapy candidate for Huntington’s disease despite the ...

The goal of gene therapy is to permanently cure hereditary diseases. One of the most promising technologies for this is the CRISPR/Cas system, colloquially known as gene scissors. These can cut and ...

The baby's disease prevented the liver from removing ammonia Half of babies with the disease die in their first week of life The baby has already shown signs of improvement The baby's disease ...

A new gene therapy can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the amount of blood the heart can pump and dramatically improves ...

A uniQure gene therapy slowed progression of Huntington’s disease by 75% after three years, statistically significant clinical trial results the company says will support plans for a regulatory ...

AVALA Hospital and Retina Associates perform Louisiana’s first ENCELTO procedure, offering new gene therapy treatment for ...

UCSF Benioff Children’s Hospital Oakland is enrolling patients in an innovative clinical trial that seeks to cure sickle cell disease. The trial is the first in the U.S. to apply non-viral CRISPR-Cas9 ...

Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall Street, and biotechnology. He is also a co-host of the weekly biotech podcast ...

DURHAM, N.C. (WTVD) -- Duke Eye Center has become the first academic medical center in the country to implant a new ...