Vincent Gaynor remembers, almost to the minute, when he realized his part in birthing the breakthrough gene therapy Zolgensma ...

AAV-Navβ1 gene therapy effectively reduces seizures and prolongs life in a mouse model of SCN1B-linked Dravet syndrome, ...

Gene therapy is a revolutionary field in modern medicine, offering transformative potential to treat and potentially cure a wide range of genetic and acquired diseases. By addressing the root ...

On this episode, an infant with a harrowing diagnosis defies all odds after receiving a $2 million genetic treatment. Then, a ...

Recent research takes aim at the a variant in gene SCN1B, which causes a severe form of developmental epileptic encephalopathy.

Gene editing is already being used to treat a handful of rare genetic diseases; however, most therapies involve breaking or ...

A £1.65 million treatment has been approved for use for some NHS patients, offering some with an inherited blood disorder hope of a cure. Campaigners reacted with joy as health officials approved a ...

The high cost of sickle cell gene therapies is pushing private insurers and Medicaid programs to develop new payment models.

Gene therapies to treat rheumatoid arthritis have evolved slowly, but one is ready to move into human trials and an ...

Berlin, Germany, and Research Triangle Park, N.C., USA, Feb. 19, 2025 (GLOBE NEWSWIRE) -- Not intended for UK Media < ...

International collaborations, refined gene-editing protocols, and targeted immunologic maneuvers keep the cancer gene therapy sector at the forefront, enabling regenerative treatments with expanded ...