Efficient manufacturing and engraftment of CCR5 gene-edited HSPCs following busulfan conditioning in nonhuman primates

Efficient manufacturing and engraftment of CCR5 gene-edited HSPCs following busulfan conditioning in nonhuman primates

Hematopoietic stem cell gene therapy has been successfully used for a number of genetic diseases and is also being explored for HIV. However, toxicity of the conditioning regimens has been a major concern. Here we compared current conditioning approaches in a clinically relevant nonhuman primate mod...

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Bibliographic Details
Main Authors: Jason Murray, Teresa Einhaus, Rasika Venkataraman, Stefan Radtke, Anjie Zhen, Mayra A. Carrillo, Scott G. Kitchen, Christopher W. Peterson, Hans-Peter Kiem
Format: Article
Language:English
Published: Elsevier 2023-09-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
hematopoietic stem cells
gene therapy
cell therapy
autologous transplant
CRISPR
conditioning
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050123001080

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http://www.sciencedirect.com/science/article/pii/S2329050123001080

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