Ontology highlight
ABSTRACT:
SUBMITTER: Ellison SM
PROVIDER: S-EPMC6479204 | biostudies-literature | 2019 Jun
REPOSITORIES: biostudies-literature
Ellison Stuart M SM Liao Aiyin A Wood Shaun S Taylor Jessica J Youshani Amir Saam AS Rowlston Sam S Parker Helen H Armant Myriam M Biffi Alessandra A Chan Lucas L Farzaneh Farzin F Wynn Rob R Jones Simon A SA Heal Paul P Gaspar H Bobby HB Bigger Brian W BW
Molecular therapy. Methods & clinical development 20190406
Hematopoietic stem cell gene therapy is a promising therapeutic strategy for the treatment of neurological disorders, since transplanted gene-corrected cells can traffic to the brain, bypassing the blood-brain barrier, to deliver therapeutic protein to the CNS. We have developed this approach for the treatment of Mucopolysaccharidosis type IIIA (MPSIIIA), a devastating lysosomal storage disease that causes progressive cognitive decline, leading to death in early adulthood. In a previous pre-clin ...[more]