recombinant adeno-associated virus-leptin

Recombinant adeno-associated virus-leptin (rAAV-leptin) is an investigational gene therapy vector designed to deliver the gene encoding leptin (LEP). Originally developed by Chiron Corporation and subsequently investigated extensively by academic institutions including the University of Florida and Oregon State University, this vector has been evaluated in preclinical models for the treatment of obesity, type 1 and type 2 diabetes, and associated metabolic and skeletal abnormalities. Depending on the route of administration, rAAV-leptin can be delivered systemically (e.g., via intramuscular injection) to achieve circulating levels of leptin, or centrally (via intracerebroventricular or direct hypothalamic microinjection) to selectively increase leptin expression in the hypothalamus. The expressed leptin protein binds to and activates the leptin receptor (LEPR), which regulates energy homeostasis, food intake, body weight, glucose metabolism, and bone density. In preclinical studies, a single administration of rAAV-leptin has demonstrated long-term efficacy in reducing body weight, adiposity, and insulin resistance, as well as correcting hyperglycemia and skeletal defects in leptin-deficient (ob/ob) and diabetic animal models.

Other names

AAV encoding leptinAAV-lepAAV-leptinrAAV encoding leptinrAAV-CBA-ObrAAV-lep