short hairpin RNA targeting EHMT2

Short hairpin RNA targeting EHMT2 is an experimental gene therapy approach being investigated for the treatment of Prader-Willi Syndrome (PWS). The therapy is being developed by Dr. Merlin Butler and his team at the University of Kansas Medical Center with funding from the Foundation for Prader-Willi Research (FPWR). The active ingredient is a short hairpin RNA (shRNA) molecule designed to silence the EHMT2 gene (also known as G9a), which encodes euchromatic histone lysine methyltransferase 2. The therapy is delivered via an adeno-associated virus vector (AAV9) for distribution throughout the central nervous system. EHMT2 is a histone methyltransferase that catalyzes the dimethylation of histone H3 lysine 9 (H3K9me2) and is involved in the epigenetic silencing of the maternal copy of PWS genes. By blocking EHMT2 function through RNA interference, the therapy aims to restore the expression of maternal PWS genes. This approach has also been studied in preclinical research settings for various cancers, including cervical, oral, lung, and pancreatic cancers.

Other names

shRNA-EHMT2shRNA-EHMT-2shRNA-EHMT 2AAV9-shRNA-EHMT2AAV-9-shRNA-EHMT2AAV 9-shRNA-EHMT2G9a-shRNAG-9a-shRNAG 9a-shRNA