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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Vydáno v:Mol Ther
Hlavní autoři: 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
Médium: Artigo
Jazyk:Inglês
Vydáno: Nature Publishing Group 2016
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Original Article
On-line přístup: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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