Self-inactivating AAV-CRISPR at different ages enables sustained amelioration of Huntington's disease deficits in BAC226Q mice - PubMed
3 hours ago
- #Gene Therapy
- #CRISPR-Cas9
- #Huntington's Disease
- Self-inactivating AAV-CRISPR system targets mutant HTT (mHTT) gene in Huntington's disease (HD).
- CRISPR-Cas9 gene editing successfully eliminates mHTT gene, reducing protein and aggregation in BAC226Q mice.
- Significant long-term improvements in neuropathology, motor deficits, weight loss, and lifespan observed.
- Effective at various disease stages—before, at, and after symptom onset.
- Self-inactivating system enhances safety by preventing prolonged Cas9 activity and off-target effects.
- Proof-of-concept supports gene editing as a viable therapeutic approach for HD.