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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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Detalles Bibliográficos
Autores principales: 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: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2018
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6023838/
https://www.ncbi.nlm.nih.gov/pubmed/30195800
http://dx.doi.org/10.1016/j.omtn.2018.04.017