In a landmark continuing collaboration, Answer ALS and Cedars-Sinai have announced the completed availability of the largest amyotrophic lateral sclerosis (ALS) patient-based induced pluripotent stem ...

It’s been a year since the gene-editing treatment Casgevy was approved for sickle cell disease and a related blood disorder.

The use of biologic and targeted therapies for children with juvenile idiopathic arthritis (JIA) surpassed more typical therapies in recent years, according to researchers.

StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models.

Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is a big problem: size.