Additional evidence sources:
- Gene Therapy for Neurodegeneration — Therapeutic approach
- Base Editing Therapy — Therapeutic modality
- AAV Delivery Methods — Vector choice
- Preclinical Validation: Test base editing efficiency in APOE4 knock-in neuronal models
- Delivery Optimization: Compare AAV versus lipid nanoparticle delivery for brain penetration
- Safety Assessment: Evaluate off-target editing and immune responses in relevant models
- Phase 1 (0-6 months): In vitro validation of base editing constructs in cell models
- Phase 2 (6-18 months): Preclinical efficacy and toxicity studies in animal models
- Phase 3 (18-36 months): IND-enabling studies and regulatory consultations