Preclinical evaluation for engraftment of CD34+ cells gene-edited at the sickle cell disease locus in xenograft mouse and non-human primate models

Preclinical evaluation for engraftment of CD34+ cells gene-edited at the sickle cell disease locus in xenograft mouse and non-human primate models

Summary: Sickle cell disease (SCD) is caused by a 20A > T mutation in the β-globin gene. Genome-editing technologies have the potential to correct the SCD mutation in hematopoietic stem cells (HSCs), producing adult hemoglobin while simultaneously eliminating sickle hemoglobin. Here, we developed...

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Bibliographic Details
Main Authors: Naoya Uchida, Linhong Li, Tina Nassehi, Claire M. Drysdale, Morgan Yapundich, Jackson Gamer, Juan J. Haro-Mora, Selami Demirci, Alexis Leonard, Aylin C. Bonifacino, Allen E. Krouse, N. Seth Linde, Cornell Allen, Madhusudan V. Peshwa, Suk See De Ravin, Robert E. Donahue, Harry L. Malech, John F. Tisdale
Format: Article
Language:English
Published: Elsevier 2021-04-01
Series:Cell Reports Medicine
Subjects:
genome editing
CRISPR/Cas9
sickle cell disease
hematopoietic stem cell
transplantation
electroporation
Online Access:http://www.sciencedirect.com/science/article/pii/S266637912100063X

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