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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...

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Detalles Bibliográficos
Autores principales:	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.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2019
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6479204/ https://www.ncbi.nlm.nih.gov/pubmed/31044143 http://dx.doi.org/10.1016/j.omtm.2019.04.001

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