Chantez Sanford Jr., 24, of Southfield, says his life has been transformed after undergoing Lyfgenia gene therapy at Children ...

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

At just 24, Chantez "CJ" Sanford is quietly making Michigan medical history. He is the first person in the state to receive ...

Daniel Cressy says today is a life-changing day. The man in his 20s received a gene therapy transfusion Wednesday at Manning ...

The last available hemophilia gene therapy is temporarily unavailable, according to CSL Behring.  | The company is working with regulators to ensure stable ongoing supply for the one-time gene therapy ...

March 11 (Reuters) - Regenxbio said on Wednesday that interim data from a early-to-mid stage study of its experimental gene therapy in patients with Duchenne muscular dystrophy showed continued ...

Enh3ance trial results indicate DTX301 reduces ammonia levels by 18% and supports dietary liberalization in patients with OTC deficiency.

Shares of Sarepta Therapeutics SRPT rose about 2% yesterday after it announced that screening and enrollment are underway in ...

Mayo Clinic researchers used milk-based nanoparticles to deliver siRNA into bile duct cancer cells. This targeted therapy ...

Forbes contributors publish independent expert analyses and insights. A patient in a late-stage gene editing trial for a rare heart condition died from fatal liver complications after receiving an ...