AAV-PHP.eB:Con-Syn::Cas9-mWPRE

AAV-PHP.eB:Con-Syn::Cas9-mWPRE is an experimental gene therapy construct designed for the treatment of familial Alzheimer's disease (FAD). It utilizes a modified adeno-associated virus (AAV) capsid, PHP.eB, which is engineered for efficient crossing of the blood-brain barrier and widespread transduction of the central nervous system following intravenous administration. The vector carries a CRISPR-Cas9 system (specifically Staphylococcus aureus Cas9) and a single-guide RNA (sgRNA) targeting the Swedish mutation in the amyloid-beta precursor protein (APP) gene. By selectively disrupting the mutated APP allele, the therapy aims to reduce amyloid-beta production, alleviate plaque pathology, and improve cognitive performance. It was developed by researchers at the Hong Kong University of Science and Technology in collaboration with the California Institute of Technology.

Other names

AAV-PHP.eB-Cas9-sgAPPAAV-PHP.eB-Cas9-sgRNA