Global and Local Manipulation of DNA Repair Mechanisms to Alter Site-Specific Gene Editing Outcomes in Hematopoietic Stem Cells

Global and Local Manipulation of DNA Repair Mechanisms to Alter Site-Specific Gene Editing Outcomes in Hematopoietic Stem Cells

Monogenic disorders of the blood system have the potential to be treated by autologous stem cell transplantation of ex vivo genetically modified hematopoietic stem and progenitor cells (HSPCs). The sgRNA/Cas9 system allows for precise modification of the genome at single nucleotide resolution. Howev...

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Bibliographic Details
Main Authors: Elizabeth K. Benitez, Anastasia Lomova Kaufman, Lilibeth Cervantes, Danielle N. Clark, Paul G. Ayoub, Shantha Senadheera, Kyle Osborne, Julie M. Sanchez, Ralph Valentine Crisostomo, Xiaoyan Wang, Nina Reuven, Yosef Shaul, Roger P. Hollis, Zulema Romero, Donald B. Kohn
Format: Article
Language:English
Published: Frontiers Media S.A. 2020-12-01
Series:Frontiers in Genome Editing
Subjects:
gene editing
hematopoietic stem cells
DNA repair
Cas9
HDR
sickle cell disease
Online Access:https://www.frontiersin.org/articles/10.3389/fgeed.2020.601541/full

Internet

https://www.frontiersin.org/articles/10.3389/fgeed.2020.601541/full

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