Editing human hematopoietic stem cells: advances and challenges

被引：8
|
作者：
Bhoopalan, Senthil Velan [1 ,2 ]
Yen, Jonathan S. [1 ]
Levine, Rachel M. [1 ]
Sharma, Akshay [2 ,3 ]
机构：
[1] St Jude Childrens Res Hosp, Dept Hematol, Memphis, TN USA
[2] St Jude Childrens Res Hosp, Dept Bone Marrow Transplantat & Cellular Therapy, Memphis, TN USA
[3] St Jude Childrens Res Hosp, Dept Bone Marrow Transplantat & Cellular Therapy, 262 Danny Thomas Pl, MS1130, Memphis, TN 38105 USA
来源：
CYTOTHERAPY | 2023年 / 25卷 / 03期
关键词：
CRISPR-Cas9; hematopoietic stem cells; severe congenital neutropenia; sickle cell disease; GENE-THERAPY; ALPHA-GLOBIN; BASE EDITORS; GENOMIC DNA; TARGET; RNA; CAS9; SPECIFICITIES; COLLECTION; NUCLEASES;
D O I：
10.1016/j.jcyt.2022.08.003
中图分类号：
Q813 [细胞工程];
学科分类号：
摘要：
Genome editing of hematopoietic stem and progenitor cells is being developed for the treatment of several inherited disorders of the hematopoietic system. The adaptation of CRISPR-Cas9-based technologies to make precise CHANGES TO THE GENOME, and developments in altering the specificity and efficiency, and improving the delivery of nucleases to target cells have led to several breakthroughs. Many clinical trials are ongoing, and several pre-clinical models have been reported that would allow these genetic therapies to one day offer a potential cure to patients with diseases where limited options currently exist. However, there remain several challenges with respect to establishing safety, expanding accessibility and improving the manufacturing pro -cesses of these therapeutic products. This review focuses on some of the recent advances in the field of genome editing of hematopoietic stem and progenitor cells and illustrates the ongoing challenges.(c) 2022 International Society for Cell & Gene Therapy. Published by Elsevier Inc. All rights reserved.
引用
收藏
页码：261 / 269
页数：9
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