APOE Isoform Conversion Therapy

APOE Isoform Conversion Therapy proposes the direct in vivo conversion of the pathogenic APOE4 allele to the protective APOE3 or APOE2 sequence using base editing or prime editing CRISPR technologies. This approach addresses the root genetic cause of APOE4-associated Alzheimer's disease risk — the single nucleotide polymorphism encoding Arg112 (vs. Cys112 in APOE3) — rather than treating downstream consequences of the APOE4 protein's dysfunctional structure. **Genetic Basis of APOE4 Pathogenici...