adeno-associated viral vector serotype 3b encoding shortened human ATP7B

adeno-associated viral vector serotype 3b encoding shortened human ATP7B is an investigational gene therapy developed by Vivet Therapeutics for the treatment of Wilson's disease. Wilson's disease is a rare autosomal recessive disorder caused by mutations in the ATP7B gene, which leads to impaired biliary copper excretion and toxic copper accumulation in the liver and brain. This therapy utilizes a liver-tropic adeno-associated viral vector of serotype 3b (AAV3b) to deliver a functional, codon-optimized, and shortened version of the human ATP7B cDNA (mini-ATP7B). The transgene is engineered to be shorter than the wild-type cDNA to fit within the limited packaging capacity of}

Other names

adeno-associated viral vector serotype 3b encoding shortened human ATP7BAAV3b-mini-ATP7BAAV-3b-mini-ATP7BAAV 3b-mini-ATP7BAAV3b-hATP7B-deltaAAV-3b-hATP7B-deltaAAV 3b-hATP7B-delta