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...

Descrición completa

Gardado en:
Detalles Bibliográficos
Publicado en: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
Formato: Artigo
Idioma:Inglês
Publicado: Nature Publishing Group 2016
Assuntos:
Original Article
Acceso en liña: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
Tags: Engadir etiqueta
Sen Etiquetas, Sexa o primeiro en etiquetar este rexistro!

Títulos similares