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Front Med (Lausanne). 2021 Dec 21;8:732095. doi: 10.3389/fmed.2021.732095. eCollection 2021.
2
Gene Therapy with an Adeno-Associated Viral Vector Expressing Human Interleukin-2 Alters Immune System Homeostasis in Humanized Mice.腺相关病毒载体介导人白细胞介素-2 基因治疗改变人源化小鼠免疫系统稳态。
Hum Gene Ther. 2018 Mar;29(3):352-365. doi: 10.1089/hum.2017.072. Epub 2017 Oct 3.

本文引用的文献

1
Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial.RPE65基因突变所致儿童期失明患者对侧眼给予AAV2基因治疗的安全性及疗效持久性:一项1期随访试验
Lancet. 2016 Aug 13;388(10045):661-72. doi: 10.1016/S0140-6736(16)30371-3. Epub 2016 Jun 30.
2
Calcium upregulation by percutaneous administration of gene therapy in patients with cardiac disease (CUPID 2): a randomised, multinational, double-blind, placebo-controlled, phase 2b trial.经皮基因治疗增强心脏病患者的钙水平(CUPID 2):一项随机、多中心、双盲、安慰剂对照、2b 期试验。
Lancet. 2016 Mar 19;387(10024):1178-86. doi: 10.1016/S0140-6736(16)00082-9. Epub 2016 Jan 21.
3
Selection and evaluation of clinically relevant AAV variants in a xenograft liver model.异种移植肝模型中临床相关 AAV 变体的选择和评估。
Nature. 2014 Feb 20;506(7488):382-6. doi: 10.1038/nature12875. Epub 2013 Dec 25.
4
Biological Heart Rate Reduction Through Genetic Suppression of Gα(s) Protein in the Sinoatrial Node.通过在窦房结中遗传抑制 Gα(s) 蛋白来降低生物心率。
J Am Heart Assoc. 2012 Apr;1(2). doi: 10.1161/JAHA.111.000372. Epub 2012 Apr 24.
5
Endgame: glybera finally recommended for approval as the first gene therapy drug in the European union.大结局:Glybera最终被推荐批准为欧盟首个基因治疗药物。
Mol Ther. 2012 Oct;20(10):1831-2. doi: 10.1038/mt.2012.194.
6
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.
7
Adeno-associated virus liver transduction efficiency measured by in vivo [18F]FHBG positron emission tomography imaging in rodents and nonhuman primates.腺相关病毒肝转导效率的活体 [18F]FHBG 正电子发射断层成像在啮齿动物和非人灵长类动物中的测量。
Hum Gene Ther. 2011 Aug;22(8):999-1009. doi: 10.1089/hum.2010.190. Epub 2011 Apr 6.
8
Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver.包含新型肝脏特异性人凝血因子IX表达盒的自互补腺相关病毒载体能够高效转导小鼠和非人类灵长类动物的肝脏。
Blood. 2006 Apr 1;107(7):2653-61. doi: 10.1182/blood-2005-10-4035. Epub 2005 Dec 1.
9
Fixing the genes.修复基因。
Time. 1999 Jan 11;153(1):68-70, 73.

Do we need marker gene studies in humans to improve clinical AAV gene therapy?

作者信息

Weber T

机构信息

Department of Medicine, Division of Cardiology, Cardiovascular Research Center and Graduate School of Biomedical Sciences, Icahn School of Medicine at Mount Sinai, New York, NY, USA.

出版信息

Gene Ther. 2017 Feb;24(2):72-73. doi: 10.1038/gt.2016.84. Epub 2017 Jan 5.

DOI:10.1038/gt.2016.84
PMID:28054581
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC5862146/
Abstract
摘要