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Myelodysplasia and transgene inactivation in X-CGD-γ retroviral gene therapy: The usual suspects and new players.

作者信息

Whittaker Thomas E, Santilli Giorgia

机构信息

Infection, Immunity, and Inflammation Teaching and Research Department, Great Ormond Street Institute of Child Health, University College London, London, UK.

Infection, Immunity, and Inflammation Teaching and Research Department, Great Ormond Street Institute of Child Health, University College London, London, UK.

出版信息

Mol Ther. 2023 Dec 6;31(12):3367-3368. doi: 10.1016/j.ymthe.2023.11.007. Epub 2023 Nov 18.

DOI:10.1016/j.ymthe.2023.11.007
PMID:37980903
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC10727967/
Abstract
摘要

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本文引用的文献

1
Myelodysplasia after clonal hematopoiesis with APOBEC3-mediated CYBB inactivation in retroviral gene therapy for X-CGD.在针对X连锁慢性肉芽肿病的逆转录病毒基因治疗中，伴有APOBEC3介导的CYBB失活的克隆性造血后发生的骨髓发育异常。
Mol Ther. 2023 Dec 6;31(12):3424-3440. doi: 10.1016/j.ymthe.2023.09.004. Epub 2023 Sep 13.
2
Severe hematopoietic stem cell inflammation compromises chronic granulomatous disease gene therapy.严重的造血干细胞炎症会损害慢性肉芽肿病的基因治疗。
Cell Rep Med. 2023 Feb 21;4(2):100919. doi: 10.1016/j.xcrm.2023.100919. Epub 2023 Jan 26.
3
Lentiviral gene therapy for X-linked chronic granulomatous disease recapitulates endogenous CYBB regulation and expression.慢病毒基因治疗 X 连锁慢性肉芽肿病再现内源性 CYBB 调控和表达。
Blood. 2023 Mar 2;141(9):1007-1022. doi: 10.1182/blood.2022016074.
4
The APOBEC Protein Family: United by Structure, Divergent in Function.载脂蛋白B编辑酶催化多肽（APOBEC）蛋白家族：结构统一，功能各异。
Trends Biochem Sci. 2016 Jul;41(7):578-594. doi: 10.1016/j.tibs.2016.05.001. Epub 2016 Jun 6.
5
Hyperinflammation in patients with chronic granulomatous disease leads to impairment of hematopoietic stem cell functions.慢性肉芽肿病患者的过度炎症会导致造血干细胞功能受损。
J Allergy Clin Immunol. 2016 Jul;138(1):219-228.e9. doi: 10.1016/j.jaci.2015.11.028. Epub 2016 Feb 4.
6
Review: Aberrant EVI1 expression in acute myeloid leukaemia.综述：急性髓系白血病中EVI1的异常表达
Br J Haematol. 2016 Mar;172(6):870-8. doi: 10.1111/bjh.13898. Epub 2016 Jan 5.
7
Gene therapy for Wiskott-Aldrich syndrome--long-term efficacy and genotoxicity.Wiskott-Aldrich 综合征的基因治疗——长期疗效和遗传毒性。
Sci Transl Med. 2014 Mar 12;6(227):227ra33. doi: 10.1126/scitranslmed.3007280.
8
Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease.基因治疗慢性肉芽肿病后 EVI1 激活导致基因组不稳定和 7 号单体性骨髓增生异常。
Nat Med. 2010 Feb;16(2):198-204. doi: 10.1038/nm.2088. Epub 2010 Jan 24.
9
Reactive oxygen species act through p38 MAPK to limit the lifespan of hematopoietic stem cells.活性氧通过p38丝裂原活化蛋白激酶发挥作用，以限制造血干细胞的寿命。
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Nucleic Acids Res. 2005 Apr 4;33(6):1913-23. doi: 10.1093/nar/gki343. Print 2005.