The delivery challenge of adeno-associated virus vector-based gene therapies for neurological diseases - PubMed
4 hours ago
- #gene therapy
- #neurological diseases
- #AAV vectors
- Gene therapies hold promise for treating neurological diseases, but effective CNS delivery vehicles are needed.
- Adeno-associated viruses (AAV), especially BBB-penetrant AAV capsids, are being explored for their potential in intravenous administration.
- The natural AAV9 capsid's ability to cross the BBB has been studied, particularly in spinal muscular atrophy treatment.
- Engineering recombinant AAV (rAAV) capsids for improved brain penetrance and in vitro models for BBB crossing prediction are discussed.
- Strategies to overcome delivery limitations of existing rAAV vectors are presented.
- Clinical studies may enhance understanding of rAAV-delivered gene therapies for CNS disorders.