AAV-PHP.eB:SW1-Syn::Cas9-mWPRE

AAV-PHP.eB:SW1-Syn::Cas9-mWPRE is an experimental CRISPR-Cas9 gene therapy construct designed for the treatment of familial Alzheimer's disease (FAD). It utilizes the engineered adeno-associated virus (AAV) capsid PHP.eB, which is capable of crossing the blood-brain barrier to achieve brain-wide delivery via non-invasive intravenous administration. The vector carries a CRISPR-Cas9 system consisting of a Cas9 nuclease and a single-guide RNA (sgRNA), designated SW1, which specifically targets the Swedish mutation (KM670/671NL) in the amyloid-beta precursor protein (APP) gene. Expression of the CRISPR components is driven by the neuron-specific synapsin (Syn) promoter and enhanced by a modified Woodchuck Hepatitis Virus Posttranscriptional Regulatory Element (mWPRE). By selectively disrupting the mutated APP allele, the therapy reduces the production of amyloid-beta peptides, alleviates plaque pathology, and improves cognitive performance in transgenic mouse models.

Other names

AAV-PHP.eB-Cas9-SW1CRISPR-Cas9 APP Swedish mutation targeting vectorCRISPR-Cas-9 APP Swedish mutation targeting vectorCRISPR-Cas 9 APP Swedish mutation targeting vector