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基因疗法：永久治愈的希望。

Gene therapy: the promise of a permanent cure.

作者信息

Porada Christopher D, Stem Christopher, Almeida-Porada Graca

机构信息

Wake Forest Institute for Regenerative Medicine, 391 Technology Way, Winston-Salem, NC 27157, USA.

出版信息

N C Med J. 2013 Nov-Dec;74(6):526-9.

PMID:24316783
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC8092353/
Abstract

Gene therapy offers the possibility of a permanent cure for any of the more than 10,000 human diseases caused by a defect in a single gene. Among these diseases, the hemophilias represent an ideal target, and studies in both animals and humans have provided evidence that a permanent cure for hemophilia is within reach.

摘要

基因治疗为治愈超过10000种由单基因缺陷引起的人类疾病提供了永久治愈的可能性。在这些疾病中，血友病是一个理想的治疗靶点，对动物和人类的研究均已证明，血友病的永久治愈指日可待。

相似文献

1
Gene therapy: the promise of a permanent cure.基因疗法：永久治愈的希望。
N C Med J. 2013 Nov-Dec;74(6):526-9.
2
Gene therapy for the hemophilias.血友病的基因治疗。
Curr Opin Hematol. 1996 Sep;3(5):385-9. doi: 10.1097/00062752-199603050-00009.
3
Hemophilia clinical gene therapy: brief review.血友病临床基因治疗：简要综述。
Transl Res. 2013 Apr;161(4):307-12. doi: 10.1016/j.trsl.2012.12.016. Epub 2013 Jan 23.
4
Gene therapy for hemophilia.血友病的基因治疗。
Hematology Am Soc Hematol Educ Program. 2019 Dec 6;2019(1):1-8. doi: 10.1182/hematology.2019000007.
5
Hemophilia Gene Therapy: New Development from Bench to Bed Side.血友病基因治疗：从实验室到临床的新进展。
Curr Gene Ther. 2019;19(4):264-273. doi: 10.2174/1566523219666190924121836.
6
Gene Therapy for Hemophilia and Duchenne Muscular Dystrophy in China.中国的血友病和杜氏肌营养不良症的基因治疗。
Hum Gene Ther. 2018 Feb;29(2):146-150. doi: 10.1089/hum.2017.213.
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Gene therapy for the hemophilias.血友病的基因治疗。
Adv Vet Med. 1997;40:119-34. doi: 10.1016/s0065-3519(97)80006-7.
8
Gene therapy to cure haemophilia: Is robust scientific inquiry the missing factor?基因疗法治疗血友病：有力的科学探究是否是缺失的因素？
Haemophilia. 2020 Nov;26(6):931-933. doi: 10.1111/hae.14131. Epub 2020 Sep 10.
9
Hemophilia gene therapy comes of age.血友病基因治疗迎来新纪元。
Hematology Am Soc Hematol Educ Program. 2017 Dec 8;2017(1):587-594. doi: 10.1182/asheducation-2017.1.587.
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[Hemophilia therapy in Italy: what the near future promises.].[意大利的血友病治疗：近期前景如何。]
Recenti Prog Med. 2023 May;114(5):247-249. doi: 10.1701/4032.40071.

引用本文的文献

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Induced Pluripotent Stem Cells (iPSCs) and Gene Therapy: A New Era for the Treatment of Neurological Diseases.诱导多能干细胞 (iPSCs) 和基因治疗：治疗神经疾病的新时代。
Int J Mol Sci. 2021 Dec 20;22(24):13674. doi: 10.3390/ijms222413674.
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Investigating Optimal Autologous Cellular Platforms for Prenatal or Perinatal Factor VIII Delivery to Treat Hemophilia A.探索用于产前或围产期递送因子VIII以治疗A型血友病的最佳自体细胞平台。
Front Cell Dev Biol. 2021 Aug 10;9:678117. doi: 10.3389/fcell.2021.678117. eCollection 2021.
3
Gene Therapy for Neurodegenerative Diseases: Slowing Down the Ticking Clock.神经退行性疾病的基因治疗：放慢生命时钟
Front Neurosci. 2020 Sep 18;14:580179. doi: 10.3389/fnins.2020.580179. eCollection 2020.
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Leukemia Inhibitory Factor (LIF) Overexpression Increases the Angiogenic Potential of Bone Marrow Mesenchymal Stem/Stromal Cells.白血病抑制因子（LIF）过表达增强骨髓间充质干/基质细胞的血管生成潜能。
Front Cell Dev Biol. 2020 Aug 14;8:778. doi: 10.3389/fcell.2020.00778. eCollection 2020.
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Mechanistic Insights into Factor VIII Immune Tolerance Induction via Prenatal Cell Therapy in Hemophilia A.对血友病A中通过产前细胞疗法诱导凝血因子VIII免疫耐受的机制性见解。
Curr Stem Cell Rep. 2019 Dec;5(4):145-161. doi: 10.1007/s40778-019-00165-y. Epub 2019 Nov 20.
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Generation and characterization of transgenic mouse mesenchymal stem cell lines expressing hIGF-1 or hG-CSF.表达人胰岛素样生长因子-1（hIGF-1）或人粒细胞集落刺激因子（hG-CSF）的转基因小鼠间充质干细胞系的生成与鉴定
Cytotechnology. 2018 Apr;70(2):577-591. doi: 10.1007/s10616-017-0131-2. Epub 2017 Sep 2.
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In utero stem cell transplantation and gene therapy: rationale, history, and recent advances toward clinical application.子宫内干细胞移植和基因治疗：原理、历史和临床应用的最新进展。
Mol Ther Methods Clin Dev. 2016 Mar 30;5:16020. doi: 10.1038/mtm.2016.20. eCollection 2016.

本文引用的文献

1
The gene therapy journey for hemophilia: are we there yet?基因治疗血友病之旅：我们到了吗？
Hematology Am Soc Hematol Educ Program. 2012;2012:375-81. doi: 10.1182/asheducation-2012.1.375.
2
The efficacy and the risk of immunogenicity of FIX Padua (R338L) in hemophilia B dogs treated by AAV muscle gene therapy.用 AAV 肌肉基因疗法治疗的血友病 B 犬中 FIX Padua（R338L）的疗效和免疫原性风险。
Blood. 2012 Nov 29;120(23):4521-3. doi: 10.1182/blood-2012-06-440123. Epub 2012 Aug 23.
3
Adenovirus-associated virus vector-mediated gene transfer in hemophilia B.腺相关病毒载体介导的乙型血友病基因转移。
N Engl J Med. 2011 Dec 22;365(25):2357-65. doi: 10.1056/NEJMoa1108046. Epub 2011 Dec 10.
4
Animal models of hemophilia.血友病动物模型。
Prog Mol Biol Transl Sci. 2012;105:151-209. doi: 10.1016/B978-0-12-394596-9.00006-8.
5
Phenotypic correction of hemophilia A in sheep by postnatal intraperitoneal transplantation of FVIII-expressing MSC.通过产后腹腔内移植表达 FVIII 的 MSC 纠正绵羊血友病 A 的表型。
Exp Hematol. 2011 Dec;39(12):1124-1135.e4. doi: 10.1016/j.exphem.2011.09.001. Epub 2011 Sep 8.
6
Gene therapy for haemophilia: a long and winding road.基因治疗血友病：漫漫长路。
J Thromb Haemost. 2011 Jul;9 Suppl 1:2-11. doi: 10.1111/j.1538-7836.2011.04369.x.
7
Efficacy and safety of long-term prophylaxis in severe hemophilia A dogs following liver gene therapy using AAV vectors.AAV 载体肝基因治疗重度 A 型血友病犬的长期预防的疗效和安全性。
Mol Ther. 2011 Mar;19(3):442-9. doi: 10.1038/mt.2010.240. Epub 2010 Nov 16.
8
Hyperactive sleeping beauty transposase enables persistent phenotypic correction in mice and a canine model for hemophilia B.过表达的睡眠美人转座酶可使小鼠和犬血友病 B 模型中持续的表型纠正。
Mol Ther. 2010 Nov;18(11):1896-906. doi: 10.1038/mt.2010.169. Epub 2010 Aug 17.
9
Prevention of spontaneous bleeding in dogs with haemophilia A and haemophilia B.预防 A 型血友病和 B 型血友病犬自发性出血。
Haemophilia. 2010 May;16 Suppl 3(Suppl 3):19-23. doi: 10.1111/j.1365-2516.2010.02255.x.
10
Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B.外周经静脉注射腺相关病毒载体治疗乙型血友病的新型疗法。
Blood. 2010 Jun 10;115(23):4678-88. doi: 10.1182/blood-2009-12-261156. Epub 2010 Mar 24.