rAAV6-HGF

rAAV6-HGF is an investigational gene therapy that utilizes a recombinant adeno-associated virus serotype 6 (rAAV6) vector to deliver the human hepatocyte growth factor (HGF) gene. The therapy is designed to induce expression of HGF in target tissues, leveraging HGF’s known roles in promoting nerve regeneration, angiogenesis, and tissue repair. The primary mechanism involves the transduction of cells with the rAAV6 vector carrying the HGF gene, leading to local production of HGF protein. This approach aims to address diseases characterized by nerve or muscle degeneration or impaired tissue repair. While plasmid-based and other viral vectors delivering HGF have been studied for neuromuscular disorders such as Charcot-Marie-Tooth disease, rAAV6-HGF specifically refers to an AAV-based delivery system for this therapeutic purpose.