Pre-clinical Safety and Efficacy of Lentiviral...

Molecular Therapy — Methods & Clinical Development 2019 / 06 Vol. 13

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

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, Si
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Volume:
13
Journal:
Molecular Therapy - Methods & Clinical Development
DOI:
10.1016/j.omtm.2019.04.001
Date:
June, 2019
File:
PDF, 2.84 MB
2019
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