Pre-clinical Safety and Efficacy of Lentiviral Vector-Mediated Ex Vivo Stem Cell Gene Therapy for the Treatment of Mucopolysaccharidosis IIIA

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 th...

Deskribapen osoa

Gorde:
Xehetasun bibliografikoak
Argitaratua izan da:Mol Ther Methods Clin Dev
Egile Nagusiak: Ellison, Stuart M., Liao, Aiyin, Wood, Shaun, Taylor, Jessica, Youshani, Amir Saam, Rowlston, Sam, Parker, Helen, Armant, Myriam, Biffi, Alessandra, Chan, Lucas, Farzaneh, Farzin, Wynn, Rob, Jones, Simon A., Heal, Paul, Gaspar, H. Bobby, Bigger, Brian W.
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: American Society of Gene & Cell Therapy 2019
Gaiak:
Article
Sarrera elektronikoa:https://ncbi.nlm.nih.gov/pmc/articles/PMC6479204/
https://ncbi.nlm.nih.gov/pubmed/31044143
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2019.04.001
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