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Challenges in vector and trial design using retroviral vectors for long-term gene correction in hematopoietic stem cell gene therapy.在造血干细胞基因治疗中使用逆转录病毒载体进行长期基因校正时，载体和试验设计面临的挑战。
Mol Ther. 2012 Jun;20(6):1084-94. doi: 10.1038/mt.2012.93.
2
Biosafety considerations using gamma-retroviral vectors in gene therapy.使用γ-逆转录病毒载体进行基因治疗的生物安全考虑因素。
Curr Gene Ther. 2013 Dec;13(6):469-77. doi: 10.2174/15665232113136660004.
3
Gene therapy for adenosine deaminase deficiency.腺苷脱氨酶缺乏症的基因治疗。
Immunol Allergy Clin North Am. 2010 May;30(2):249-60. doi: 10.1016/j.iac.2010.02.003.
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Solving the problem of γ-retroviral vectors containing long terminal repeats.解决含有长末端重复序列的γ-逆转录病毒载体问题。
Mol Ther. 2011 Feb;19(2):229-31. doi: 10.1038/mt.2010.305.
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Retroviral vectors: post entry events and genomic alterations.逆转录病毒载体：进入后事件和基因组改变。
Viruses. 2011 May;3(5):429-55. doi: 10.3390/v3050429. Epub 2011 Apr 29.
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HOXB4 and retroviral vectors: adding fuel to the fire.HOXB4与逆转录病毒载体：火上浇油
J Clin Invest. 2008 Apr;118(4):1350-3. doi: 10.1172/JCI35326.
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Side effects of retroviral gene transfer into hematopoietic stem cells.逆转录病毒基因转移至造血干细胞的副作用。
Blood. 2003 Mar 15;101(6):2099-114. doi: 10.1182/blood-2002-07-2314. Epub 2003 Jan 2.
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Genotoxicity of retroviral hematopoietic stem cell gene therapy.逆转录病毒造血干细胞基因治疗的遗传毒性。
Expert Opin Biol Ther. 2011 May;11(5):581-93. doi: 10.1517/14712598.2011.562496. Epub 2011 Mar 7.
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Safety concerns related to hematopoietic stem cell gene transfer using retroviral vectors.与使用逆转录病毒载体进行造血干细胞基因转移相关的安全问题。
Curr Gene Ther. 2004 Sep;4(3):263-76. doi: 10.2174/1566523043346174.
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Retroviral vector-mediated gene transfer into hematopoietic cells: prospects and issues.逆转录病毒载体介导的基因转移至造血细胞：前景与问题
J Hematother. 1992 Summer;1(2):155-66. doi: 10.1089/scd.1.1992.1.155.

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Novel Insights into the Therapeutic Potential of Lung-Targeted Gene Transfer in the Most Common Respiratory Diseases.新型见解：肺部靶向基因转移在最常见呼吸系统疾病中的治疗潜力。
Cells. 2022 Mar 12;11(6):984. doi: 10.3390/cells11060984.
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Gene Therapy for Respiratory Diseases: Progress and a Changing Context.呼吸系统疾病的基因治疗：进展与不断变化的背景
Hum Gene Ther. 2020 Sep;31(17-18):911-916. doi: 10.1089/hum.2020.142.
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Evidence for the in vivo safety of insulated foamy viral vectors.绝缘泡沫病毒载体体内安全性的证据。
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Genome editing technologies: defining a path to clinic.基因组编辑技术：确定通往临床应用的路径。
Mol Ther. 2015 May;23(5):796-806. doi: 10.1038/mt.2015.54.
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Cell-based interventions in utero: time to reconsider.子宫内基于细胞的干预措施：是时候重新审视了。
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Lentiviral vectors for the treatment of primary immunodeficiencies.用于治疗原发性免疫缺陷的慢病毒载体。
J Inherit Metab Dis. 2014 Jul;37(4):525-33. doi: 10.1007/s10545-014-9690-y. Epub 2014 Mar 12.
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Preclinical demonstration of lentiviral vector-mediated correction of immunological and metabolic abnormalities in models of adenosine deaminase deficiency.临床前研究展示慢病毒载体介导的腺苷脱氨酶缺乏症模型免疫和代谢异常的纠正。
Mol Ther. 2014 Mar;22(3):607-622. doi: 10.1038/mt.2013.265. Epub 2013 Nov 20.
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Gene therapy on the move.基因治疗正在发展中。
EMBO Mol Med. 2013 Nov;5(11):1642-61. doi: 10.1002/emmm.201202287. Epub 2013 Sep 17.
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No impact of lentiviral transduction on hematopoietic stem/progenitor cell telomere length or gene expression in the rhesus macaque model.慢病毒转导对恒河猴模型造血干/祖细胞端粒长度或基因表达无影响。
Mol Ther. 2014 Jan;22(1):52-8. doi: 10.1038/mt.2013.168. Epub 2013 Jul 18.

本文引用的文献

1
Human T Lymphotropic Virus Type 1 (HTLV-1): Molecular Biology and Oncogenesis.人类 T 淋巴细胞白血病病毒 1 型（HTLV-1）：分子生物学与致癌性。
Viruses. 2010 Sep;2(9):2037-2077. doi: 10.3390/v2092037. Epub 2010 Sep 24.
2
Gene therapy for primary immunodeficiency.原发性免疫缺陷的基因治疗。
Curr Opin Pediatr. 2011 Dec;23(6):659-66. doi: 10.1097/MOP.0b013e32834cd67a.
3
Concise review: managing genotoxicity in the therapeutic modification of stem cells.简明综述：干细胞治疗修饰中的遗传毒性管理。
Stem Cells. 2011 Oct;29(10):1479-84. doi: 10.1002/stem.716.
4
Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction.腺苷脱氨酶缺乏症严重联合免疫缺陷症的造血干细胞基因治疗可实现长期免疫恢复和代谢纠正。
Sci Transl Med. 2011 Aug 24;3(97):97ra80. doi: 10.1126/scitranslmed.3002716.
5
Long-term persistence of a polyclonal T cell repertoire after gene therapy for X-linked severe combined immunodeficiency.基因治疗 X 连锁重症联合免疫缺陷后多克隆 T 细胞 repertoire 的长期持久性。
Sci Transl Med. 2011 Aug 24;3(97):97ra79. doi: 10.1126/scitranslmed.3002715.
6
Transposon-mediated gene transfer into adult and induced pluripotent stem cells.转座子介导的基因转移到成年和诱导多能干细胞。
Curr Gene Ther. 2011 Oct;11(5):406-13. doi: 10.2174/156652311797415836.
7
Site-specific integration and tailoring of cassette design for sustainable gene transfer.位点特异性整合和盒式设计的定制化用于可持续基因转移。
Nat Methods. 2011 Aug 21;8(10):861-9. doi: 10.1038/nmeth.1674.
8
CTF/NF1 transcription factors act as potent genetic insulators for integrating gene transfer vectors.CTF/NF1 转录因子可作为整合基因转移载体的有效遗传绝缘子。
Gene Ther. 2012 Jan;19(1):15-24. doi: 10.1038/gt.2011.70. Epub 2011 May 12.
9
3'UTR-truncated Hmga2 cDNA causes MPN-like hematopoiesis by conferring a clonal growth advantage at the level of HSC in mice.3'UTR 截短的 Hmga2 cDNA 通过在 HSC 水平赋予克隆生长优势导致类似于 MPN 的造血。
Blood. 2011 Jun 2;117(22):5860-9. doi: 10.1182/blood-2011-02-334425. Epub 2011 Apr 1.
10
Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection.慢病毒载体在临床前模型和临床试验中的常见整合位点反映了良性的整合偏向，而不是致癌性选择。
Blood. 2011 May 19;117(20):5332-9. doi: 10.1182/blood-2010-09-306761. Epub 2011 Mar 14.

Challenges in vector and trial design using retroviral vectors for long-term gene correction in hematopoietic stem cell gene therapy.

作者信息

Corrigan-Curay Jacqueline, Cohen-Haguenauer Odile, O'Reilly Marina, Ross Susan R, Fan Hung, Rosenberg Naomi, Somia Nikunj, King Nancy, Friedmann Theodore, Dunbar Cynthia, Aiuti Alessandro, Naldini Luigi, Baum Christopher, von Kalle Christof, Kiem Hans-Peter, Montini Eugenio, Bushman Frederic, Sorrentino Brian P, Carrondo Manuel, Malech Harry, Gahrton Gösta, Shapiro Robyn, Wolff Linda, Rosenthal Eugene, Jambou Robert, Zaia John, Kohn Donald B

机构信息

Biotechnology Activities, National Institutes of Health, Bethesda, Maryland, USA.

出版信息

Mol Ther. 2012 Jun;20(6):1084-94. doi: 10.1038/mt.2012.93.

DOI:10.1038/mt.2012.93
PMID:22652996
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC3369284/
Abstract
摘要