AAV8-UCP1-Rb1

AAV8-UCP1-Rb1 is a preclinical gene therapy candidate designed for the treatment of obesity and related metabolic disorders. Developed by researchers at Shanghai Ninth People's Hospital and Shanghai Jiao Tong University School of Medicine, the therapy utilizes an adeno-associated virus serotype 8 (AAV8) vector to deliver the retinoblastoma 1 (Rb1) gene. Expression of the transgene is driven by the brown adipose tissue (BAT)-specific uncoupling protein 1 (UCP1) promoter, restricting its activity to thermogenic adipocytes. Mechanistically, the overexpression of the RB protein induces G1 cell-cycle arrest, which triggers the activation of the p38-mTORC1 signaling cascade and drives RB-dependent mitochondrial remodeling and thermogenesis. In diet-induced obesity (DIO) mouse models, targeted delivery of AAV8-UCP1-Rb1 robustly stimulates BAT thermogenesis and increases systemic energy expenditure, offering a potential therapeutic strategy for obesity.

Other names

AAV8-UCP1-Rb1AAV-8-UCP1-Rb1AAV 8-UCP1-Rb1