rAAV Gene Therapy in a Canavan's Disease Mouse Model Reveals Immune Impairments and an Extended Pathology Beyond the Central Nervous System

Aspartoacylase (AspA) gene mutations cause the pediatric lethal neurodegenerative Canavan disease (CD). There is emerging promise of successful gene therapy for CD using recombinant adeno-associated viruses (rAAVs). Here, we report an intracerebroventricularly delivered AspA gene therapy regime usin...

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Bibliografiske detaljer
Udgivet i:Mol Ther
Main Authors: Ahmed, Seemin Seher, Schattgen, Stefan A, Frakes, Ashley E, Sikoglu, Elif M, Su, Qin, Li, Jia, Hampton, Thomas G, Denninger, Andrew R, Kirschner, Daniel A, Kaspar, Brian, Matalon, Reuben, Gao, Guangping
Format: Artigo
Sprog:Inglês
Udgivet: Nature Publishing Group 2016
Fag:
Original Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4923332/
https://ncbi.nlm.nih.gov/pubmed/27039844
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2016.68
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