CNS-restricted Transduction and CRISPR/Cas9-mediated Gene Deletion with an Engineered AAV Vector

• Main Authors: Giridhar Murlidharan (Author), Kensuke Sakamoto (Author), Lavanya Rao (Author), Travis Corriher (Author), Dan Wang (Author), Guangping Gao (Author), Patrick Sullivan (Author), Aravind Asokan (Author)
• Format: Book
• Published: Elsevier, 2016-01-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Gene therapy using recombinant adeno-associated viral (AAV) vectors is emerging as a promising approach to treat central nervous system disorders such as Spinal muscular atrophy, Batten, Parkinson and Alzheimer disease amongst others. A critical remaining challenge for central nervous system-targete...