A Single Intravenous rAAV Injection as Late as P20 Achieves Efficacious and Sustained CNS Gene Therapy in Canavan Mice

Canavan's disease (CD) is a fatal pediatric leukodystrophy caused by mutations in aspartoacylase (AspA) gene. Currently, there is no effective treatment for CD; however, gene therapy is an attractive approach to ameliorate the disease. Here, we studied progressive neuropathology and gene therap...

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Main Authors: Ahmed, Seemin Seher, Li, Huapeng, Cao, Chunyan, Sikoglu, Elif M, Denninger, Andrew R, Su, Qin, Eaton, Samuel, Liso Navarro, Ana A, Xie, Jun, Szucs, Sylvia, Zhang, Hongwei, Moore, Constance, Kirschner, Daniel A, Seyfried, Thomas N, Flotte, Terence R, Matalon, Reuben, Gao, Guangping
Format: Artigo
Jezik:Inglês
Izdano: Nature Publishing Group 2013
Teme:
Original Article
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC3863789/
https://ncbi.nlm.nih.gov/pubmed/23817205
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2013.138
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