plasmid DNA encoding human ATP7B

Plasmid DNA encoding human ATP7B is a non-viral gene therapy candidate being developed for the treatment of Wilson Disease (WD). Unlike adeno-associated virus (AAV) vectors, which are limited by a 4.4kb packaging capacity, this plasmid-based approach allows for the delivery of the full-length 4.4kb ATP7B cDNA. The therapy is delivered via hydrodynamic injection through the biliary system using endoscopic retrograde cholangiography (ERCP), a technique designed to bypass the limitations of systemic delivery and AAV-related immunogenicity. Preclinical studies in human-sized pig models have demonstrated efficient hepatocyte transfection and the feasibility of redosing. The program is a collaboration involving HydroGene Therapeutics and the Mayo Clinic.

Other names

pDNA-hATP7BpDNA-hATP-7BpDNA-hATP 7BhATP7B plasmidhATP-7B plasmidhATP 7B plasmidfull-length human ATP7B plasmid DNA