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
相关论文
共 50 条
  • [21] Editing an α-globin enhancer in primary human hematopoietic stem cells as a treatment for β-thalassemia
    Sachith Mettananda
    Chris A. Fisher
    Deborah Hay
    Mohsin Badat
    Lynn Quek
    Kevin Clark
    Philip Hublitz
    Damien Downes
    Jon Kerry
    Matthew Gosden
    Jelena Telenius
    Jackie A. Sloane-Stanley
    Paula Faustino
    Andreia Coelho
    Jessica Doondeea
    Batchimeg Usukhbayar
    Paul Sopp
    Jacqueline A. Sharpe
    Jim R. Hughes
    Paresh Vyas
    Richard J. Gibbons
    Douglas R. Higgs
    Nature Communications, 8
  • [22] Gene Editing of Human Hematopoietic Stem and Progenitor Cells: Promise and Potential Hurdles
    Yu, Kyung-Rok
    Natanson, Hannah
    Dunbar, Cynthia E.
    HUMAN GENE THERAPY, 2016, 27 (10) : 729 - 740
  • [23] Current approaches and potential challenges in the delivery of gene editing cargos into hematopoietic stem and progenitor cells
    Murugesan, Ramya
    Karuppusamy, Karthik V.
    Marepally, Srujan
    Thangavel, Saravanabhavan
    FRONTIERS IN GENOME EDITING, 2023, 5
  • [24] Gene Editing in Human Pluripotent Stem Cells: Recent Advances for Clinical Therapies
    Sisli, Hatice Burcu
    Hayal, Taha Bartu
    Seckin, Selin
    Senkal, Selinay
    Kiratli, Binnur
    Sahin, Fikrettin
    Dogan, Aysegul
    CELL BIOLOGY AND TRANSLATIONAL MEDICINE, VOL 7: STEM CELLS AND THERAPY: EMERGING APPROACHES, 2020, 1237 : 17 - 28
  • [25] Hematopoietic stem cells: Challenges to expectations
    Morrison, SJ
    Wright, DE
    Cheshier, SH
    Weissman, IL
    CURRENT OPINION IN IMMUNOLOGY, 1997, 9 (02) : 216 - 221
  • [26] TALEN®-Based Gene Editing in Hematopoietic Stem Cells
    Busser, Brian W.
    Temburni, Sonal
    Boyne, Alex
    Juillerat, Alexandre
    Poirot, Laurent
    Duchateau, Philippe
    BLOOD, 2018, 132
  • [27] GENOME EDITING OF HEMATOPOIETIC STEM CELLS TO ENGINEER BLOOD
    Porteus, Matthew
    EXPERIMENTAL HEMATOLOGY, 2022, 111 : S29 - S29
  • [28] Therapy Development by Genome Editing of Hematopoietic Stem Cells
    Koniali, Lola
    Lederer, Carsten W.
    Kleanthous, Marina
    CELLS, 2021, 10 (06)
  • [29] Immunogenetics Targeted "Genome Editing" of hematopoietic Stem Cells
    Lorenz, Judith
    TRANSFUSIONSMEDIZIN, 2014, 4 (04) : 172 - 173
  • [30] Converting human pluripotent stem cells into β-cells: recent advances and future challenges
    Mayhew, Christopher N.
    Wells, James M.
    CURRENT OPINION IN ORGAN TRANSPLANTATION, 2010, 15 (01) : 54 - 60
12345