AAV9-LSP-UFµDys2

AAV9-LSP-UFµDys2 is an investigational adeno-associated virus (AAV) serotype 9 gene therapy vector developed by researchers at the University of Florida for the treatment of Duchenne Muscular Dystrophy (DMD). The construct is specifically designed to address the immune challenges associated with dystrophin replacement therapy. It utilizes a liver-specific promoter (LSP) to drive the expression of a truncated micro-dystrophin transgene (µDys2) primarily in hepatocytes. The therapeutic rationale for this approach is to induce hepatic-mediated immune tolerance to the micro-dystrophin protein. By presenting the transgene product in the tolerogenic environment of the liver, the therapy aims to prevent or mitigate the development of anti-dystrophin T-cell responses, which can otherwise lead to the rejection of micro-dystrophin expressed in skeletal and cardiac muscle. This vector is typically studied as part of a strategy to enhance the safety and long-term persistence of muscle-targeted gene therapies.

Other names

AAV9-LSP-micro-dystrophinAAV-9-LSP-micro-dystrophinAAV 9-LSP-micro-dystrophinUF-micro-dystrophin-2UF-micro-dystrophin2UF-micro-dystrophin 2