Long before there was Sarepta's gene therapy for muscular dystrophy, there was Donavon Decker and his offer to test a gene ...

STAT reporters discuss more turmoil in the FDA's regulation of gene therapies and a model of funding academic research with private capital.

How Does Gene Therapy for Hemophilia Work? Gene therapy, which is now FDA-approved for hemophilia A and hemophilia B, deliver genes for factor VIII or IX via adeno-associated viruses.

Gene therapy appears to be a promising approach for a subset of genetic deafness, although challenges remain with development and manufacturing.

Lentiviral vector (LV)–based gene therapy using CD34+ hematopoietic stem cells (HSCs) demonstrated stable factor VIII expression in severe hemophilia A patients, with higher vector copy numbers ...

Researchers have developed a gene therapy that significantly slowed motor function loss in preclinical models of amyotrophic lateral sclerosis (ALS), offering new hope for treating the devastating ...

This Gene Therapy Maker Refuses to Halt Shipments After Patient Deaths The FDA called for Sarepta Therapeutics to stop shipping its muscular dystrophy gene therapy, but the company said no.

Youth with congenital deafness treated with gene therapy showed better progress on certain hearing measures than counterparts treated with cochlear implantation in a cohort study from China. Gene ...

The Revell family hoped a gene therapy could buy time for their sons, who have a rare and fatal disease. After two patients died, the drug’s manufacturer halted dosing under pressure from the FDA.