AAV9-ADAR2

AAV9-ADAR2 is an adeno-associated virus (AAV) serotype 9 vector-based gene therapy designed to deliver the ADAR2 (ADARB1) gene to motor neurons. In sporadic amyotrophic lateral sclerosis (sALS), the loss of ADAR2 activity leads to defective A-to-I RNA editing of the GluA2 subunit of AMPA receptors at the Q/R site. This defect results in calcium-permeable AMPA receptors, which cause excitotoxic motor neuron death. By restoring ADAR2 expression, the therapy aims to normalize GluA2 editing, prevent calcium influx, and halt the progression of motor neuron degeneration. It was primarily developed by researchers at the University of Tokyo and GeneCare Research Institute.

Other names

ADAR2 gene therapyADAR-2 gene therapyADAR 2 gene therapyADARB1 gene therapyADARB-1 gene therapyADARB 1 gene therapy