AAV-CYP46A1

AAV-CYP46A1 is an adeno-associated virus (AAV) gene therapy designed to deliver the *CYP46A1* gene to the central nervous system. The *CYP46A1* gene encodes cholesterol 24-hydroxylase, an enzyme that converts cholesterol into 24-hydroxycholesterol, which can cross the blood-brain barrier. By overexpressing this enzyme, the therapy aims to restore brain cholesterol homeostasis and promote the clearance of toxic proteins, which are key pathological features in various neurodegenerative diseases. Research has been primarily led by the Paris Brain Institute (Institut du Cerveau) and has shown potential in preclinical models of Alzheimer's disease, Huntington's disease, Parkinson's disease, amyotrophic lateral sclerosis (ALS), and spinocerebellar ataxia type 3 (SCA3).

Other names

AAV9-CYP46A1AAV-9-CYP46A1AAV 9-CYP46A1