artificial microRNA targeting tau

Artificial microRNAs targeting tau (Tau-miRNAs) are experimental RNA-based gene therapy candidates designed to reduce tau protein levels in the brain for the treatment of tauopathies, including Alzheimer's disease and other neurodegenerative disorders. The approach has been developed by researchers at CONICET (Argentina), with inventor Maria Elena Avale holding a provisional patent (US 63/359,519; 2023). The artificial microRNAs target the human MAPT (microtubule-associated protein tau) transcript to lower tau protein synthesis through the endogenous RNA interference pathway. The modality is a gene therapy utilizing artificial microRNAs delivered via viral vectors (lentiviral or AAV vectors) for local, targeted expression in vulnerable brain regions. Separately, Voyager Therapeutics has developed its own tau silencing gene therapy program using a BBB-penetrant AAV containing primary artificial microRNA targeting tau, which the company advanced into late research with plans to file an IND application in 2026. Preclinical studies have shown that these artificial microRNAs can prevent tau pathology accumulation, improve cognitive function, and restore neuronal firing properties in mouse models of tauopathy.

Other names

anti-tau artificial microRNAsanti-tau amiRNAs