CRISPR/Cas9 gene editing in nonhuman primate model demonstrates no intrinsic clonal expansion of PNH HSPCs.
作者信息
Shin Tae-Hoon, Baek Eun Jung, Corat Marcus A F, Chen Shirley, Metais Jean-Yves, AlJanahi Aisha A, Zhou Yifan, Donahue Robert E, Yu Kyung-Rok, Dunbar Cynthia E
机构信息
Translational Stem Cell Biology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD.
Department of Laboratory Medicine, College of Medicine, Hanyang University, Seoul, Republic of Korea.
出版信息
Blood. 2019 Jun 6;133(23):2542-2545. doi: 10.1182/blood.2019000800. Epub 2019 Apr 19.
Abstract
摘要
相似文献
1
CRISPR/Cas9 gene editing in nonhuman primate model demonstrates no intrinsic clonal expansion of PNH HSPCs.
Blood. 2019 Jun 6;133(23):2542-2545. doi: 10.1182/blood.2019000800. Epub 2019 Apr 19.
2
Paroxysmal nocturnal hemoglobinuria: stem cells and clonality.
Hematology Am Soc Hematol Educ Program. 2008:111-5. doi: 10.1182/asheducation-2008.1.111.
3
Immune pathogenesis of paroxysmal nocturnal hemoglobinuria.
Int J Hematol. 2006 Aug;84(2):113-7. doi: 10.1532/IJH97.06144.
4
A cohort study of the nature of paroxysmal nocturnal hemoglobinuria clones and PIG-A mutations in patients with aplastic anemia.
Eur J Haematol. 2006 Jun;76(6):502-9. doi: 10.1111/j.0902-4441.2005.t01-1-EJH2467.x. Epub 2006 Mar 9.
5
Highly Efficient Genome Editing of Murine and Human Hematopoietic Progenitor Cells by CRISPR/Cas9.
Cell Rep. 2016 Oct 25;17(5):1453-1461. doi: 10.1016/j.celrep.2016.09.092.
6
PIG-A mutations in paroxysmal nocturnal hemoglobinuria and in normal hematopoiesis.
Leuk Lymphoma. 2006 Jul;47(7):1215-21. doi: 10.1080/10428190600555520.
7
[Paroxysmal nocturnal hemoglobinuria: mechanism of clonal expansion].
Rinsho Ketsueki. 2005 May;46(5):317-23.
8
Murine embryonic stem cells without pig-a gene activity are competent for hematopoiesis with the PNH phenotype but not for clonal expansion.
J Clin Invest. 1997 Sep 1;100(5):1028-36. doi: 10.1172/JCI119613.
9
The molecular basis of paroxysmal nocturnal hemoglobinuria.
Haematologica. 2000 Jan;85(1):82-7.
10
[Advances of studies on pathogenesis and management of paroxysmal nocturnal hemoglobinuria].
Zhongguo Shi Yan Xue Ye Xue Za Zhi. 2013 Apr;21(2):530-5. doi: 10.7534/j.issn.1009-2137.2013.02.058.
引用本文的文献
1
Impact of CRISPR/HDR editing versus lentiviral transduction on long-term engraftment and clonal dynamics of HSPCs in rhesus macaques.
Cell Stem Cell. 2024 Apr 4;31(4):455-466.e4. doi: 10.1016/j.stem.2024.02.010. Epub 2024 Mar 19.
2
Case report: Immune pressure on hematopoietic stem cells can drastically expand glycosylphosphatidylinositol-deficient clones in paroxysmal nocturnal hemoglobinuria.
Front Immunol. 2024 Jan 15;14:1329403. doi: 10.3389/fimmu.2023.1329403. eCollection 2023.
3
Long noncoding RNA FAM157C contributes to clonal proliferation in paroxysmal nocturnal hemoglobinuria.
Ann Hematol. 2023 Feb;102(2):299-309. doi: 10.1007/s00277-022-05055-8. Epub 2023 Jan 6.
4
A macaque clonal hematopoiesis model demonstrates expansion of TET2-disrupted clones and utility for testing interventions.
Blood. 2022 Oct 20;140(16):1774-1789. doi: 10.1182/blood.2021014875.
5
Modeling genetic diseases in nonhuman primates through embryonic and germline modification: Considerations and challenges.
Sci Transl Med. 2022 Mar 2;14(634):eabf4879. doi: 10.1126/scitranslmed.abf4879.
6
Insights Into the Emergence of Paroxysmal Nocturnal Hemoglobinuria.
Front Immunol. 2022 Jan 28;12:830172. doi: 10.3389/fimmu.2021.830172. eCollection 2021.
7
A Pig-a conditional knock-out mice model mediated by Vav-iCre: stable GPI-deficient and mild hemolysis.
Exp Hematol Oncol. 2022 Jan 15;11(1):1. doi: 10.1186/s40164-022-00254-5.
8
When does a PNH clone have clinical significance?
Hematology Am Soc Hematol Educ Program. 2021 Dec 10;2021(1):143-152. doi: 10.1182/hematology.2021000245.
9
Understanding and overcoming adverse consequences of genome editing on hematopoietic stem and progenitor cells.
Mol Ther. 2021 Nov 3;29(11):3205-3218. doi: 10.1016/j.ymthe.2021.09.001. Epub 2021 Sep 10.
10
Prediction and validation of hematopoietic stem and progenitor cell off-target editing in transplanted rhesus macaques.
Mol Ther. 2022 Jan 5;30(1):209-222. doi: 10.1016/j.ymthe.2021.06.016. Epub 2021 Jun 24.
本文引用的文献
1
Repair of double-strand breaks induced by CRISPR-Cas9 leads to large deletions and complex rearrangements.
Nat Biotechnol. 2018 Sep;36(8):765-771. doi: 10.1038/nbt.4192. Epub 2018 Jul 16.
2
Genetic Inactivation of CD33 in Hematopoietic Stem Cells to Enable CAR T Cell Immunotherapy for Acute Myeloid Leukemia.
Cell. 2018 May 31;173(6):1439-1453.e19. doi: 10.1016/j.cell.2018.05.013.
3
Quantitative stability of hematopoietic stem and progenitor cell clonal output in rhesus macaques receiving transplants.
Blood. 2017 Mar 16;129(11):1448-1457. doi: 10.1182/blood-2016-07-728691. Epub 2017 Jan 13.
4
Gene Editing of Human Hematopoietic Stem and Progenitor Cells: Promise and Potential Hurdles.
Hum Gene Ther. 2016 Oct;27(10):729-740. doi: 10.1089/hum.2016.107. Epub 2016 Aug 2.
5
Therapeutic genome editing: prospects and challenges.
Nat Med. 2015 Feb;21(2):121-31. doi: 10.1038/nm.3793.
6
Paroxysmal nocturnal hemoglobinuria.
Blood. 2014 Oct 30;124(18):2804-11. doi: 10.1182/blood-2014-02-522128. Epub 2014 Sep 18.
7
Clonal tracking of rhesus macaque hematopoiesis highlights a distinct lineage origin for natural killer cells.
Cell Stem Cell. 2014 Apr 3;14(4):486-499. doi: 10.1016/j.stem.2014.01.020.
8
Hematopoietic stem cell gene therapy:assessing the relevance of preclinical models.
Semin Hematol. 2013 Apr;50(2):101-30. doi: 10.1053/j.seminhematol.2013.03.025.
9
Genomic safe harbors permit high β-globin transgene expression in thalassemia induced pluripotent stem cells.
Nat Biotechnol. 2011 Jan;29(1):73-8. doi: 10.1038/nbt.1717. Epub 2010 Dec 12.
10
Phenotypic and functional characterization of a mouse model of targeted Pig-a deletion in hematopoietic cells.
Haematologica. 2010 Feb;95(2):214-23. doi: 10.3324/haematol.2009.011650. Epub 2009 Aug 13.
Zotero 插件