Ontology highlight
ABSTRACT:
SUBMITTER: Griffin TA
PROVIDER: S-EPMC4437470 | biostudies-literature | 2015 May
REPOSITORIES: biostudies-literature
Griffin Tagan A TA Anderson Hayley C HC Wolfe John H JH
Stem cell reports 20150409 5
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 storage disease, Sly disease. Human Sly disease fibroblasts were reprogrammed into iPSCs, differentiated into a stable and expandable population of NSCs, genetically corrected with a transposon vector, ...[more]