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

The new method is designed to focus specifically on pain-related signals, without interfering with normal activity in other ...

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

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 ...

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

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 ...

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 ...

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 ...

Abu Dhabi achieved a medical milestone on January 5, 2026, with the UAE's first gene-therapy injection for inherited blood disorders. CASGEVY, utilizing CRISPR-Cas9, corrects faulty DNA in conditions ...