Learn how a one-time intravenous infusion of the high dose of gene therapy extended the survival of patients with spinal muscular atrophy type 1 (SMA1) in a Phase 1 clinical trial.

Modern medicine constantly searches for safer and more efficient ways to treat genetic disorders. One cutting-edge solution that has emerged is the use of non-viral vectors.

For the first time, we are witnessing therapies that can fundamentally alter the course of inherited disease lifelong. The most recent breakthrough describes treating inherited disease in infants ...

Scientists report that gene therapy restored at least some hearing and speech for five out of six children with a rare form of genetic deafness.

Experimental gene therapy studied in both China and the United States has allowed several children born with inherited deafness to hear. On Tuesday, the Children's Hospital of Philadelphia ...

Three decades after its first, faltering steps in humans, gene therapy is emerging as a treatment option for a small but growing number of diseases. Although the concept faced scientific and ...

Opal Sandy, who was born deaf because of a rare genetic condition, and can now hear unaided for the first time after receiving ground-breaking gene therapy at 11-months-old, at her home in Eynsham ...

The FDA approves the first of two gene therapies this month to treat sickle cell disease. Though expensive and difficult, it promises a better life.

Gene therapy has come a long way since its first human proof-of-concept trials in the 1990s. The approach—which involves fixing or replacing a disease-causing gene or changing its activity—has ...

Researchers at University of California San Diego School of Medicine have developed a gene therapy for Alzheimer’s disease that could help protect the brain from damage and preserve cognitive function ...