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.

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

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

Drug delivery researchers have vastly improved the potential of genetic therapies by overcoming the challenge of consistently getting genes and gene-editing tools where they need to be within cells.

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 ...

The last available hemophilia gene therapy is temporarily unavailable, according to CSL Behring. Hemgenix, CSL’s one-time gene therapy for patients with hemophilia B, is experiencing a “temporary ...

Researchers at the Yong Loo Lin School of Medicine, National University of Singapore (NUS Medicine) have developed a revolutionary new method to improve compact gene-editing tools known as base ...

A pioneering gene therapy could help treat a rare seizure disorder called Dravet syndrome, according to new clinical trial results. The drug, called zorevunersen, holds promise, especially for people ...

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

As a potential biomarker of treatment response, ctDNA and utDNA analyses were performed before systemic therapy, at restaging, and for responders at the beginning of adjuvant nivolumab. ctDNA was ...