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 ...

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 ...

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

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 ...

As newborn screening and rapid DNA sequencing become routine, we are poised to catch and treat inherited diseases at their earliest stages. Today, we can intervene in the first days or weeks of life.

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 ...

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

A growing body of evidence suggests that a single gene, APOE, may play a far more dominant role in Alzheimer’s disease than ...

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