Increasing CRISPR Efficiency and Measuring Its Specificity in HSPCs Using a Clinically Relevant System

Increasing CRISPR Efficiency and Measuring Its Specificity in HSPCs Using a Clinically Relevant System

Genome editing of human cluster of differentiation 34+ (CD34+) hematopoietic stem and progenitor cells (HSPCs) holds great therapeutic potential. This study aimed to optimize on-target, ex vivo genome editing using the CRISPR-Cas9 system in CD34+ HSPCs and to create a clear workflow for precise iden...

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Bibliographic Details
Main Authors: Jenny Shapiro, Ortal Iancu, Ashley M. Jacobi, Matthew S. McNeill, Rolf Turk, Garrett R. Rettig, Ido Amit, Adi Tovin-Recht, Zohar Yakhini, Mark A. Behlke, Ayal Hendel
Format: Article
Language:English
Published: Elsevier 2020-06-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
genome editing
CRISPR-Cas9
CD34+ hematopoietic stem and progenitor cells
chemically modified guide RNAs
off-target sites
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050120300875

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http://www.sciencedirect.com/science/article/pii/S2329050120300875

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