AAV-ATXN2 RNAi

AAV-ATXN2 RNAi is a preclinical gene therapy candidate designed to treat TDP-43 proteinopathies, including amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The approach utilizes a peptide-modified adeno-associated virus 9 (PM-AAV9) vector, engineered for superior central nervous system (CNS) targeting, to deliver microRNAs (miRNAs) that silence the ATXN2 (Ataxin-2) gene via RNA interference (RNAi). Reduction of Ataxin-2 protein has been shown to mitigate TDP-43 pathology, a hallmark of neurodegeneration in ALS and FTD. Preclinical studies in mouse models have demonstrated that a single intracerebroventricular (ICV) injection provides sustained, widespread ATXN2 knockdown throughout the CNS, leading to improved survival, motor strength, and normalization of sleep architecture.

Other names

PM-AAV9-ATXN2-miRNAPM-AAV-9-ATXN2-miRNAPM-AAV 9-ATXN2-miRNAAAV-mediated ATXN2 knockdown