体内造血干细胞转导
In Vivo Hematopoietic Stem Cell Transduction.
作者信息
Richter Maximilian, Stone Daniel, Miao Carol, Humbert Olivier, Kiem Hans-Peter, Papayannopoulou Thalia, Lieber André
机构信息
Division of Medical Genetics, University of Washington, 1705 NE Pacific Street, Seattle, WA 98195, USA.
Vaccine and Infectious Disease Division, Fred Hutchinson Cancer Research Center, 1100 Fairview Avenue N, Seattle, WA 98109, USA.
出版信息
Hematol Oncol Clin North Am. 2017 Oct;31(5):771-785. doi: 10.1016/j.hoc.2017.06.001.
Abstract
Current protocols for hematopoietic stem cell (HSC) gene therapy, involving the transplantation of ex vivo lentivirus vector-transduced HSCs into myeloablated recipients, are complex and not without risk for the patient. In vivo HSC gene therapy can be achieved by the direct modification of HSCs in the bone marrow after intraosseous injection of gene delivery vectors. A recently developed approach involves the mobilization of HSCs from the bone marrow into peripheral the blood circulation, intravenous vector injection, and re-engraftment of genetically modified HSCs in the bone marrow. We provide examples for in vivo HSC gene therapy and discuss advantages and disadvantages.
摘要
目前的造血干细胞（HSC）基因治疗方案，包括将体外慢病毒载体转导的造血干细胞移植到接受过骨髓清除的受体中，过程复杂且对患者存在风险。体内造血干细胞基因治疗可通过在骨内注射基因递送载体后直接修饰骨髓中的造血干细胞来实现。最近开发的一种方法涉及将造血干细胞从骨髓动员到外周血液循环中，进行静脉内载体注射，然后将基因修饰的造血干细胞重新植入骨髓。我们提供了体内造血干细胞基因治疗的实例，并讨论了其优缺点。
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Biol Blood Marrow Transplant. 2017 Feb;23(2):247-254. doi: 10.1016/j.bbmt.2016.10.025. Epub 2016 Nov 2.
2
In vivo correction of anaemia in β-thalassemic mice by γPNA-mediated gene editing with nanoparticle delivery.经纳米颗粒递送的 γPNA 介导的基因编辑实现β-地中海贫血小鼠体内贫血纠正。
Nat Commun. 2016 Oct 26;7:13304. doi: 10.1038/ncomms13304.
3
Adenovirus sensing by the immune system.免疫系统对腺病毒的感知。
Curr Opin Virol. 2016 Dec;21:109-113. doi: 10.1016/j.coviro.2016.08.017. Epub 2016 Sep 14.
4
A comparison of foamy and lentiviral vector genotoxicity in SCID-repopulating cells shows foamy vectors are less prone to clonal dominance.在 SCID 重建细胞中比较泡沫和慢病毒载体的遗传毒性，结果表明泡沫载体不易发生克隆优势。
Mol Ther Methods Clin Dev. 2016 Aug 17;3:16048. doi: 10.1038/mtm.2016.48. eCollection 2016.
5
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Blood. 2016 Nov 3;128(18):2206-2217. doi: 10.1182/blood-2016-04-711580. Epub 2016 Aug 23.
6
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J Immunol Methods. 2016 Jul;434:1-8. doi: 10.1016/j.jim.2016.03.005. Epub 2016 Apr 3.
7
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Nat Commun. 2016 Mar 15;7:11007. doi: 10.1038/ncomms11007.
8
Targeted gene addition in human CD34(+) hematopoietic cells for correction of X-linked chronic granulomatous disease.在人类CD34(+)造血细胞中进行靶向基因添加以纠正X连锁慢性肉芽肿病。
Nat Biotechnol. 2016 Apr;34(4):424-9. doi: 10.1038/nbt.3513. Epub 2016 Mar 7.
9
Homology-driven genome editing in hematopoietic stem and progenitor cells using ZFN mRNA and AAV6 donors.使用锌指核酸酶（ZFN）mRNA和腺相关病毒6型（AAV6）供体在造血干细胞和祖细胞中进行同源性驱动的基因组编辑。
Nat Biotechnol. 2015 Dec;33(12):1256-1263. doi: 10.1038/nbt.3408. Epub 2015 Nov 9.
10
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Mol Ther. 2015 Apr;23(4):617-26. doi: 10.1038/mt.2015.20. Epub 2015 Feb 6.
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