Topic: therapeutic gene sequences

Technological advances have made Gene therapy a promising therapeutic strategy for a wide range of neurodegenerative diseases. Adeno-associated viruses (AAVs) have demonstrated significant potential as safe and effective vectors for the delivery of therapeutic genes to the CNS. Despite the tremendous promises of AAV-based gene therapies, the pathway to develop these therapies from bench to clinic remains complex and arduous. In response to these challenges, we introduce the BrainVector project, a comprehensive cross-disciplinary platform that streamlines the pre-clinical development of AAV-bas