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...

ver descrição completa

Na minha lista:
Detalhes bibliográficos
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
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2013
Assuntos:
Original Article
Acesso em linha: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
Tags: Adicionar Tag
Sem tags, seja o primeiro a adicionar uma tag!

Registos relacionados