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Ex Vivo Gene Therapy Using Patient iPSC-Derived NSCs Reverses Pathology in the Brain of a Homologous Mouse Model

Neural stem cell (NSC) transplantation is a promising strategy for delivering therapeutic proteins in the brain. We evaluated a complete process of ex vivo gene therapy using human induced pluripotent stem cell (iPSC)-derived NSC transplants in a well-characterized mouse model of a human lysosomal s...

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Detalles Bibliográficos
Autores principales: Griffin, Tagan A., Anderson, Hayley C., Wolfe, John H.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2015
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4437470/
https://www.ncbi.nlm.nih.gov/pubmed/25866157
http://dx.doi.org/10.1016/j.stemcr.2015.02.022