adaptamer-regulated FGF21

Adaptamer-regulated FGF21 is an experimental gene therapy developed by researchers at the University of California San Diego. It employs a compact RNA switch called an 'adaptamer' (ADAR modulatable aptamer) to provide small-molecule-controlled expression of Fibroblast Growth Factor 21 (FGF21). The system works by incorporating a tetracycline-responsive aptamer into the mRNA of the FGF21 transgene; upon tetracycline administration, the aptamer recruits endogenous ADAR (Adenosine Deaminase Acting on RNA) to convert an internal stop codon into a sense codon, thereby triggering protein translation. This cleavage-free, post-transcriptional mechanism allows for precise, reversible, and dose-dependent modulation of FGF21 levels. In preclinical studies using diet-induced obese mice, AAV-mediated delivery of this construct successfully reversed weight gain, improved glucose tolerance, and reduced hepatic steatosis, highlighting its potential for treating metabolic diseases.

Other names

AAV-FGF21 adaptamerAAV-FGF-21 adaptamerAAV-FGF 21 adaptamerinducible AAV-FGF21