AAV-hfCas13Y-gMAPT

AAV-hfCas13Y-gMAPT is an experimental gene therapy designed to treat Alzheimer's disease by targeting tau pathology. Developed by HuidaGene Therapeutics in collaboration with the Shanghai Institute of Materia Medica, the therapy utilizes a high-fidelity CRISPR-Cas13Y (hfCas13Y) system delivered via an adeno-associated virus (AAV) vector. Unlike traditional CRISPR-Cas9 systems that edit DNA, Cas13Y is an RNA-guided RNA endonuclease that specifically degrades target mRNA—in this case, the transcript for microtubule-associated protein tau (MAPT). By reducing MAPT mRNA levels, the therapy aims to decrease the production of tau protein and its subsequent aggregation into neurofibrillary tangles. Preclinical data in PS19 mouse models have shown that hippocampal or intracerebroventricular administration of AAV-hfCas13Y-gMAPT leads to significant reductions in MAPT expression and phosphorylated tau levels, resulting in improved cognitive and memory functions for at least nine months post-injection.

Other names

AAV-CRISPR/hfCas13Y-gMAPT