AAV6-U6-pol3-RNAi

AAV6-U6-pol3-RNAi is an experimental gene therapy construct designed for the treatment of myotonic dystrophy type 1 (DM1). It utilizes an adeno-associated virus serotype 6 (AAV6) vector to deliver an RNA interference (RNAi) sequence, typically in the form of a short hairpin RNA (shRNA), under the control of a U6 RNA polymerase III (pol 3) promoter. The therapy is engineered to target the expanded CUG repeat-containing DMPK mRNA, which is the primary driver of DM1 pathology. By silencing the toxic expanded transcripts, the therapy aims to prevent the sequestration of the splicing factor MBNL1, thereby restoring normal alternative splicing of cellular pre-mRNAs and alleviating muscle dysfunction. Developed by researchers at the University of Washington, this construct has been evaluated in the HSALR mouse model, demonstrating efficacy in reversing aberrant splicing patterns both as a monotherapy and as part of a dual-vector approach combined with MBNL1 protein supplementation.

Other names

AAV6-U6-RNAiAAV-6-U6-RNAiAAV 6-U6-RNAiAAV6-delivered U6 pol 3 expressed HSA RNAiAAV-6-delivered U6 pol 3 expressed HSA RNAiAAV 6-delivered U6 pol 3 expressed HSA RNAi