A shot in the bone corrects a genetic disease.
作者信息
Brown Brian D
机构信息
Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York, New York USA.
出版信息
Mol Ther. 2015 Apr;23(4):614-5. doi: 10.1038/mt.2015.38.
Abstract
摘要
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本文引用的文献
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Rapamycin relieves lentiviral vector transduction resistance in human and mouse hematopoietic stem cells.雷帕霉素可缓解人源和小鼠造血干细胞中的慢病毒载体转导抗性。
Blood. 2014 Aug 7;124(6):913-23. doi: 10.1182/blood-2013-12-546218. Epub 2014 Jun 9.
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Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy.慢病毒造血干细胞基因治疗有益于脑苷脂沉积病。
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Gene therapy for hemophilia.血友病的基因治疗。
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A TLR and non-TLR mediated innate response to lentiviruses restricts hepatocyte entry and can be ameliorated by pharmacological blockade.TLR 和非 TLR 介导的先天免疫反应限制了慢病毒进入肝细胞，可通过药物阻断加以改善。
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Lentivirus-mediated platelet gene therapy of murine hemophilia A with pre-existing anti-factor VIII immunity.慢病毒介导的血小板基因治疗对具有抗因子 VIII 免疫的小鼠血友病 A。
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