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Gene delivery GAD 500 autoantigen by AAV serotype 1 prevented diabetes in NOD mice: transduction efficiency do not play important roles.通过1型腺相关病毒(AAV)载体递送谷氨酸脱羧酶500自身抗原可预防非肥胖糖尿病(NOD)小鼠患糖尿病:转导效率并非起重要作用。
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Expression of the human multidrug resistance and glucocerebrosidase cDNAs from adeno-associated vectors: efficient promoter activity of AAV sequences and in vivo delivery via liposomes.腺相关载体表达人多药耐药和葡萄糖脑苷脂酶cDNA:腺相关病毒序列的高效启动子活性及通过脂质体进行体内递送
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Defective adenoassociated viral-mediated transfection of insulin gene by direct injection into liver parenchyma decreases blood glucose of diabetic mice.通过直接注射到肝实质中,腺相关病毒介导的胰岛素基因转染缺陷可降低糖尿病小鼠的血糖。
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本文引用的文献

1
Rescue of Hearing by Gene Delivery to Inner-Ear Hair Cells Using Exosome-Associated AAV.利用外泌体相关腺相关病毒向内耳毛细胞进行基因传递来恢复听力
Mol Ther. 2017 Feb 1;25(2):379-391. doi: 10.1016/j.ymthe.2016.12.010. Epub 2017 Jan 9.
2
Application of Mouse Models to Research in Hearing and Balance.小鼠模型在听力与平衡研究中的应用
J Assoc Res Otolaryngol. 2016 Dec;17(6):493-523. doi: 10.1007/s10162-016-0589-1. Epub 2016 Oct 17.
3
On the Distant Horizon--Medical Therapy for Sensorineural Hearing Loss.遥远的前景——感音神经性听力损失的医学治疗
Otolaryngol Clin North Am. 2015 Dec;48(6):1149-65. doi: 10.1016/j.otc.2015.07.012. Epub 2015 Sep 26.
4
Tmc gene therapy restores auditory function in deaf mice.Tmc基因疗法可恢复耳聋小鼠的听觉功能。
Sci Transl Med. 2015 Jul 8;7(295):295ra108. doi: 10.1126/scitranslmed.aab1996.
5
Surgical method for virally mediated gene delivery to the mouse inner ear through the round window membrane.通过圆窗膜将病毒介导的基因递送至小鼠内耳的手术方法。
J Vis Exp. 2015 Mar 16(97):52187. doi: 10.3791/52187.
6
Treating hearing disorders with cell and gene therapy.用细胞和基因疗法治疗听力障碍。
J Neural Eng. 2014 Dec;11(6):065001. doi: 10.1088/1741-2560/11/6/065001.
7
Virally expressed connexin26 restores gap junction function in the cochlea of conditional Gjb2 knockout mice.病毒表达的连接蛋白26可恢复条件性Gjb2基因敲除小鼠耳蜗中的缝隙连接功能。
Gene Ther. 2014 Jan;21(1):71-80. doi: 10.1038/gt.2013.59. Epub 2013 Nov 14.
8
A missense mutation in the previously undescribed gene Tmhs underlies deafness in hurry-scurry (hscy) mice.此前未被描述的基因Tmhs中的一个错义突变是匆匆忙忙(hscy)小鼠耳聋的根本原因。
Proc Natl Acad Sci U S A. 2005 May 31;102(22):7894-9. doi: 10.1073/pnas.0500760102. Epub 2005 May 19.
9
Expression of adeno-associated virus integrated transgene within the mammalian vestibular organs.腺相关病毒整合转基因在哺乳动物前庭器官中的表达。
Am J Otol. 1998 May;19(3):390-5.

It's All in the Delivery: Improving AAV Transfection Efficiency with Exosomes.

作者信息

Martin Donna M, Raphael Yehoash

机构信息

Departments of Pediatrics and Human Genetics, The University of Michigan, 1150 W. Medical Ctr. Dr., 3520D MSRB-I, Ann Arbor, MI 48109-5652.

Kresge Hearing Research Institute, Department of Otolaryngology - Head and Neck Surgery, The University of Michigan, 9220B MSRB-III, Ann Arbor, MI 48109-5648.

出版信息

Mol Ther. 2017 Feb 1;25(2):309-311. doi: 10.1016/j.ymthe.2017.01.004. Epub 2017 Jan 23.

DOI:10.1016/j.ymthe.2017.01.004
PMID:28126363
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC5368841/
Abstract
摘要