Priming Human Repopulating Hematopoietic Stem and Progenitor Cells for Cas9/sgRNA Gene Targeting

Engineered nuclease-mediated gene targeting through homologous recombination (HR) in hematopoietic stem and progenitor cells (HSPCs) has the potential to treat a variety of genetic hematologic and immunologic disorders. Here, we identify critical parameters to reproducibly achieve high frequencies o...

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Detalhes bibliográficos
Publicado no:Mol Ther Nucleic Acids
Main Authors: Charlesworth, Carsten T., Camarena, Joab, Cromer, M. Kyle, Vaidyanathan, Sriram, Bak, Rasmus O., Carte, Jason M., Potter, Jason, Dever, Daniel P., Porteus, Matthew H.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2018
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6023838/
https://ncbi.nlm.nih.gov/pubmed/30195800
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2018.04.017
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