Gene therapy treatments for rare diseases are being developed, but getting them out of the lab has proved challenging.

A patient death in a CRISPR trial halts gene editing studies, raising critical safety questions. What this means for the ...

Cell and gene therapies use the body's building blocks - cells and genetic material - to boost the immune system or correct ...

People looking to lose weight and lower their blood sugar may someday be able to get a single injection that turns their cells into tiny factories churning out a protein that is essentially the active ...

Gene therapy is becoming a powerful way to treat challenging diseases that don’t respond to traditional treatments, and researchers now report the first success in modifying genes to slow Huntington’s ...

A preclinical study uncovered a new gene therapy that targets pain centers in the brain while eliminating the risk of ...

Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing ...

There is no cure for the rare disease Hereditary Spastic Paraplegia (HSP), but researchers from Drexel University's College of Medicine and the UMass Chan Medical School have achieved ...

Novartis has received approval from the U.S. Food and Drug Administration for Itvisma, a treatment for spinal muscular atrophy. Dr. John Day is professor of neurology and pediatrics, director of the ...