AAV-mediated NEK1 gene augmentation therapy

AAV-mediated NEK1 gene augmentation therapy is an experimental gene therapy designed to treat amyotrophic lateral sclerosis (ALS) caused by loss-of-function mutations in the NIMA-related kinase 1 (NEK1) gene. NEK1 mutations account for approximately 2-3% of ALS cases. The therapy utilizes adeno-associated virus (AAV) vectors to deliver a functional copy of the human NEK1 gene to the central nervous system. Preclinical studies in NEK1 knockout mouse models have demonstrated that intracerebroventricular (ICV) administration of these AAV constructs can restore NEK1 protein levels in the brain. The program is being developed by researchers at the University of Massachusetts Chan Medical School in collaboration with The Jackson Laboratory.

Other names

NEK1 gene augmentation therapyNEK-1 gene augmentation therapyNEK 1 gene augmentation therapyAAV-NEK1AAV-NEK-1AAV-NEK 1