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.
Abstract
摘要
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本文引用的文献
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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。
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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。
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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。
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