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

• المؤلفون الرئيسيون: Giridhar Murlidharan (مؤلف), Kensuke Sakamoto (مؤلف), Lavanya Rao (مؤلف), Travis Corriher (مؤلف), Dan Wang (مؤلف), Guangping Gao (مؤلف), Patrick Sullivan (مؤلف), Aravind Asokan (مؤلف)
• التنسيق: كتاب
• منشور في: Elsevier, 2016-01-01T00:00:00Z.
• الموضوعات: article
• الوصول للمادة أونلاين: 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...