AAV-RIP-shWnt5a

AAV-RIP-shWnt5a is an experimental adeno-associated virus (AAV) vector-based gene therapy designed to knock down the expression of Wnt family member 5A (Wnt5a) specifically in pancreatic beta cells. The construct utilizes the rat insulin promoter (RIP) to drive the expression of a short hairpin RNA (shRNA) targeting Wnt5a. In the context of type 2 diabetes mellitus (T2DM), Wnt5a is upregulated and contributes to beta-cell dysfunction by suppressing TFEB-dependent lysosomal biogenesis and autophagy. By silencing Wnt5a, this therapeutic candidate aims to restore lysosomal function, enhance autophagic flux, and preserve beta-cell homeostasis, thereby improving glucose tolerance and potentially slowing diabetes progression.