Efficient Ex Vivo Engineering and Expansion of Highly Purified Human Hematopoietic Stem and Progenitor Cell Populations for Gene Therapy

Ex vivo gene therapy based on CD34(+) hematopoietic stem cells (HSCs) has shown promising results in clinical trials, but genetic engineering to high levels and in large scale remains challenging. We devised a sorting strategy that captures more than 90% of HSC activity in less than 10% of mobilized...

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Udgivet i:Stem Cell Reports
Main Authors: Zonari, Erika, Desantis, Giacomo, Petrillo, Carolina, Boccalatte, Francesco E., Lidonnici, Maria Rosa, Kajaste-Rudnitski, Anna, Aiuti, Alessandro, Ferrari, Giuliana, Naldini, Luigi, Gentner, Bernhard
Format: Artigo
Sprog:Inglês
Udgivet: Elsevier 2017
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5390102/
https://ncbi.nlm.nih.gov/pubmed/28330619
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.stemcr.2017.02.010
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