AAV-BEST1

**AAV-BEST1** is an experimental **AAV-mediated BEST1 gene replacement therapy** being investigated for inherited retinal bestrophinopathies such as **Best vitelliform macular dystrophy** and **autosomal recessive bestrophinopathy**. The approach uses an adeno-associated viral vector, reported in the literature particularly as **AAV2/2**, to deliver a functional **BEST1** transgene to **retinal pigment epithelium** cells with the goal of restoring bestrophin-1 activity and correcting disease caused by loss-of-function or pathogenic BEST1 variants. It appears to be a **preclinical research/program designation** rather than a branded commercial product, and has been associated with development efforts including work attributed to **Nightstar Therapeutics**.

Other names

BEST1 gene therapyBEST-1 gene therapyBEST 1 gene therapyAAV2/2-BEST1