Conference: AAIC 2026 | Dates: July 12-15, 2026 | Location: Excel London, UK
Gene therapy represents a transformative approach to treating Alzheimer's disease, with the potential to deliver disease-modifying payloads directly to the brain. AAIC 2026 features the latest advances in viral vector delivery, gene editing, and RNA-targeted approaches for neurodegenerative diseases.
- Serotype selection: CNS-tropic AAV capsids
- AAV9 and AAVrh.10: Most common in CNS applications
- Novel capsids: Engineered variants with enhanced brain targeting
- Delivery routes: Intrathecal, intravenous, and direct brain injection
- Blood-brain barrier crossing: Strategies to overcome BBB
- Promoter selection: Cell-type specific expression
- Dose optimization: Balancing efficacy and toxicity
- Repeat dosing challenges: Immune response considerations
- APP gene silencing: siRNA and antisense approaches
- BACE1 knock-down: Reducing amyloid precursor cleavage
- Anti-amyloid antibody genes: In vivo antibody production
- MAPT gene modulation: Reducing tau expression
- Tau kinase inhibitors: Gene-based delivery
- Anti-tau antibody genes: Continuous antibody production
- BDNF delivery: Brain-derived neurotrophic factor
- GDNF delivery: Glial cell line-derived neurotrophic factor
- Nrf2 gene therapy: Antioxidant response activation
- CLU (Clusterin): Enhancing amyloid clearance
- Gene knock-out: Targeting risk genes (e.g., APOE4)
- Gene knock-in: Protective allele introduction
- Base editing: Precise single-nucleotide modifications
- Prime editing: Broader sequence modifications
- AAV-Cas9 delivery: In vivo editing systems
- Lipid nanoparticle delivery: Non-viral alternatives
- Ex vivo editing: Cell-based approaches
- APP-targeting ASOs: Reducing amyloid production
- MAPT-targeting ASOs: Reducing tau expression
- SOD1-targeting ASOs: For ALS/AD overlap
- Viral-vector delivered siRNA: Long-term silencing
- Non-viral nanocarriers: Safety advantages
- Target cell selection: Specific neuronal populations
- AAV-BDNF trials: Phase 1/2 results expected
- AAV-GDNF trials: Parkinsonism studies with AD relevance
- AAV-anti-amyloid trials: Novel antibody gene delivery
- APP-targeting ASOs: Safety and efficacy data
- Tau-targeting ASOs: Initial dose-escalation results
- Combination approaches: Multiple target strategies
- Long-term follow-up: Duration requirements
- Pediatric considerations: Early intervention potential
- Somatic vs. germline editing: Ethical frameworks
- "Gene Therapy for Alzheimer's Disease: Where Are We?"
- "AAV Vectors for CNS Disorders: Engineering the Perfect Capsid"
- "RNA-Targeted Approaches in Neurodegeneration"
- "Designing Gene Therapy Clinical Trials for AD"
- "Biomarkers for Gene Therapy Response"
- "Manufacturing Challenges for AAV Production"
- Gene Therapy for Neurodegeneration
- AAV Vector Biology
- CRISPR Gene Editing
- BDNF Signaling Pathway
- RNA Interference Mechanisms