An expert involved in the first successful gene therapy trial for Huntington's Disease discusses the trial results and ...

Eli Lilly and Regeneron are leading the push to treat congenital deafness with gene therapies, seeking a piece of a potential ...

The Barbara Ann Karmanos Cancer Institute has become the first and only independent cancer center in the U.S. to provide a ...

With promise to slow aging and cure some types of cancer, a new gene therapy presents cost and access hurdles for CT patients ...

Gene therapy represents one of medicine’s most ambitious attempts to treat disease at its root cause by altering the genetic code itself. The approach works by recovering the functions of critical ...

SEATTLE — Some of our most challenging health conditions result from genetic disorders like sickle cell disease and certain cancers. Gene therapy is revolutionizing how they're treated, and the Fred ...

The last available hemophilia gene therapy is temporarily unavailable, according to CSL Behring.  | The company is working ...

A genetic variant carried by at least one-fifth of the population may do far more than raise the odds of developing Alzheimer ...

She has a genetic condition that affects motor nerve cells in the spinal cord, causing progressive muscle weakness. Read more ...

Onasemnogene abeparvovec gene therapy post-nusinersen or risdiplam showed meaningful motor improvements in children with SMA, with a manageable safety profile. The study cohort included older, heavier ...

A new method for safely inserting large chunks of DNA into genomes has now measured up in mice, potentially paving the way ...