Ad5.pAPOE.PE-Ch

Ad5.pAPOE.PE-Ch is an E1-E3-deleted adenovirus serotype 5 (Ad5) vector-based gene therapy designed for in vivo prime editing. Developed by researchers at Weill Cornell Medicine, the vector delivers a complete prime editing cassette, including a Cas9 nickase-reverse transcriptase fusion protein and a prime editing guide RNA (pegRNA), under the control of a liver-specific APOE promoter. The therapy aims to treat Alzheimer's disease by introducing the protective Christchurch (R136S) mutation into the human apolipoprotein E (APOE) gene, specifically targeting individuals with the APOE4 risk variant. By utilizing an Ad5 vector, the system achieves rapid, transient expression of the editing machinery, which is sufficient for genomic modification while minimizing the risks associated with long-term expression of CRISPR-based components.