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Mol Ther. 2024 Oct 2;32(10):3201-3202. doi: 10.1016/j.ymthe.2024.09.014. Epub 2024 Sep 17.
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Gene therapy through autologous transplantation of gene-modified hematopoietic stem cells.通过基因修饰的造血干细胞自体移植进行基因治疗。
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Promises and Challenges in Hematopoietic Stem Cell Gene Therapy.造血干细胞基因治疗的前景与挑战。
Hum Gene Ther. 2017 Oct;28(10):782-799. doi: 10.1089/hum.2017.141.
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Human haematopoietic progenitors as a target for gene therapy.作为基因治疗靶点的人类造血祖细胞
Scott Med J. 1993 Oct;38(5):131-3. doi: 10.1177/003693309303800501.
6
Clinical hematopoietic stem cell-based gene therapy.基于临床造血干细胞的基因治疗。
Mol Ther. 2025 Jun 4;33(6):2663-2678. doi: 10.1016/j.ymthe.2025.04.029. Epub 2025 Apr 24.
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Preclinical lentiviral hematopoietic stem cell gene therapy corrects Pompe disease-related muscle and neurological manifestations.临床前慢病毒造血干细胞基因治疗纠正庞贝病相关的肌肉和神经表现。
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本文引用的文献

1
Secondary failure of lentiviral vector gene therapy in a cerebral adrenoleukodystrophy patient with an ABCD1 whole-gene deletion.ABCD1 全基因缺失的脑肾上腺脑白质营养不良患者慢病毒载体基因治疗的继发性失败。
Mol Ther. 2024 Oct 2;32(10):3313-3317. doi: 10.1016/j.ymthe.2024.08.005. Epub 2024 Aug 5.
2
Immunogenicity and toxicity of AAV gene therapy.AAV 基因治疗的免疫原性和毒性。
Front Immunol. 2022 Aug 12;13:975803. doi: 10.3389/fimmu.2022.975803. eCollection 2022.
3
Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access.慢病毒造血干细胞基因治疗早发性异染性脑白质营养不良：1/2 期非随机、开放标签、单臂临床试验及扩大使用的长期结果。
Lancet. 2022 Jan 22;399(10322):372-383. doi: 10.1016/S0140-6736(21)02017-1.
4
Complexity of immune responses to AAV transgene products - Example of factor IX.对腺相关病毒转基因产物免疫反应的复杂性 - 因子 IX 为例。
Cell Immunol. 2019 Aug;342:103658. doi: 10.1016/j.cellimm.2017.05.006. Epub 2017 May 29.
5
Changes in the incidence, patterns and outcomes of graft failure following hematopoietic stem cell transplantation for Hurler syndrome.黏多糖贮积症I型患者造血干细胞移植后移植物失败的发生率、模式及结局变化
Bone Marrow Transplant. 2017 Jun;52(6):846-853. doi: 10.1038/bmt.2017.5. Epub 2017 Feb 20.
6
Total body irradiation must be delivered at high dose for efficient engraftment and tolerance in a rhesus stem cell gene therapy model.全身照射必须以高剂量给予，以在恒河猴干细胞基因治疗模型中实现有效的植入和耐受。
Mol Ther Methods Clin Dev. 2016 Sep 14;3:16059. doi: 10.1038/mtm.2016.59. eCollection 2016.
7
Novel approaches and mechanisms in hematopoietic stem cell gene therapy.造血干细胞基因治疗的新方法和机制
Discov Med. 2014 Apr;17(94):207-15.
8
Hematopoietic stem cell transplantation improves the high incidence of neutralizing allo-antibodies observed in Hurler's syndrome after pharmacological enzyme replacement therapy.造血干细胞移植改善了在进行药物性酶替代治疗后，在Hurler综合征中观察到的高频率中和性同种抗体的情况。
Haematologica. 2012 Sep;97(9):1320-8. doi: 10.3324/haematol.2011.058644. Epub 2012 Feb 27.
9
Retrovirus gene therapy for X-linked chronic granulomatous disease can achieve stable long-term correction of oxidase activity in peripheral blood neutrophils.逆转录病毒基因治疗 X 连锁慢性肉芽肿病可实现外周血中性粒细胞氧化酶活性的稳定长期纠正。
Blood. 2010 Jan 28;115(4):783-91. doi: 10.1182/blood-2009-05-222760. Epub 2009 Dec 1.
10
Induction of transgene-specific immunological tolerance in myeloablated nonhuman primates using lentivirally transduced CD34+ progenitor cells.使用慢病毒转导的CD34+祖细胞在骨髓消融的非人灵长类动物中诱导转基因特异性免疫耐受。
Mol Ther. 2003 Dec;8(6):981-91. doi: 10.1016/j.ymthe.2003.08.020.

Immune responses to cells and proteins after hematopoietic stem cell gene therapy for inherited diseases: A cause for concern.

作者信息

Jeffreys Nathan, Wynn Robert

机构信息

Blood and Marrow Transplant Programme, Royal Manchester Children's Hospital, Manchester, UK.

Blood and Marrow Transplant Programme, Royal Manchester Children's Hospital, Manchester, UK.

出版信息

Mol Ther. 2024 Oct 2;32(10):3201-3202. doi: 10.1016/j.ymthe.2024.09.014. Epub 2024 Sep 17.

DOI:10.1016/j.ymthe.2024.09.014
PMID:39293430
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11489541/
Abstract
摘要