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

• Asıl Yazarlar: Giridhar Murlidharan (Yazar), Kensuke Sakamoto (Yazar), Lavanya Rao (Yazar), Travis Corriher (Yazar), Dan Wang (Yazar), Guangping Gao (Yazar), Patrick Sullivan (Yazar), Aravind Asokan (Yazar)
• Materyal Türü: Kitap
• Baskı/Yayın Bilgisi: Elsevier, 2016-01-01T00:00:00Z.
• Konular: article
• Online Erişim: 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...