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本文引用的文献

1
Cochlear gene therapy with ancestral AAV in adult mice: complete transduction of inner hair cells without cochlear dysfunction.成年小鼠耳蜗内源性 AAV 基因治疗:内毛细胞完全转导而不损害耳蜗功能。
Sci Rep. 2017 Apr 3;7:45524. doi: 10.1038/srep45524.
2
Gene Therapy Restores Balance and Auditory Functions in a Mouse Model of Usher Syndrome.基因疗法可恢复乌舍尔综合征小鼠模型的平衡和听觉功能。
Mol Ther. 2017 Mar 1;25(3):780-791. doi: 10.1016/j.ymthe.2017.01.007. Epub 2017 Feb 21.
3
Gene therapy restores auditory and vestibular function in a mouse model of Usher syndrome type 1c.基因治疗可恢复1c型Usher综合征小鼠模型的听觉和前庭功能。
Nat Biotechnol. 2017 Mar;35(3):264-272. doi: 10.1038/nbt.3801. Epub 2017 Feb 6.
4
RNA Interference Prevents Autosomal-Dominant Hearing Loss.RNA干扰可预防常染色体显性遗传性听力损失。
Am J Hum Genet. 2016 Jun 2;98(6):1101-1113. doi: 10.1016/j.ajhg.2016.03.028. Epub 2016 May 26.
5
Gene Therapy Restores Hair Cell Stereocilia Morphology in Inner Ears of Deaf Whirler Mice.基因疗法恢复了耳聋Whirler小鼠内耳毛细胞静纤毛的形态。
Mol Ther. 2016 Feb;24(1):17-25. doi: 10.1038/mt.2015.150. Epub 2015 Aug 26.
6
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.
7
Gene therapy for sensorineural hearing loss.感音神经性听力损失的基因治疗
Ear Hear. 2015 Jan;36(1):1-7. doi: 10.1097/AUD.0000000000000088.
8
Systemic lipopolysaccharide compromises the blood-labyrinth barrier and increases entry of serum fluorescein into the perilymph.全身性脂多糖会损害血迷路屏障,并增加血清荧光素进入外淋巴的量。
J Assoc Res Otolaryngol. 2014 Oct;15(5):707-19. doi: 10.1007/s10162-014-0476-6. Epub 2014 Jun 21.
9
Rescue of hearing and vestibular function by antisense oligonucleotides in a mouse model of human deafness.反义寡核苷酸在人类耳聋小鼠模型中对听力和前庭功能的挽救作用。
Nat Med. 2013 Mar;19(3):345-50. doi: 10.1038/nm.3106. Epub 2013 Feb 4.
10
Restoration of hearing in the VGLUT3 knockout mouse using virally mediated gene therapy.利用病毒介导的基因治疗恢复 VGLUT3 敲除小鼠的听力。
Neuron. 2012 Jul 26;75(2):283-93. doi: 10.1016/j.neuron.2012.05.019.

新生小鼠内耳基因递送的后半规管途径

Posterior Semicircular Canal Approach for Inner Ear Gene Delivery in Neonatal Mouse.

作者信息

Isgrig Kevin, Chien Wade W

机构信息

National Institute on Deafness and Other Communication Disorders, National Institutes of Health.

National Institute on Deafness and Other Communication Disorders, National Institutes of Health; Department of Otolaryngology-Head & Neck Surgery, Johns Hopkins School of Medicine;

出版信息

J Vis Exp. 2018 Mar 2(133):56648. doi: 10.3791/56648.

DOI:10.3791/56648
PMID:29553564
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC5931426/
Abstract

Inner ear gene therapy offers great promise as a potential treatment for hearing loss and dizziness. One of the critical determinants of the success of inner ear gene therapy is to find a delivery method which results in consistent transduction efficiency of targeted cell types while minimizing hearing loss. In this study, we describe the posterior semicircular canal approach as a viable method for inner ear gene delivery in neonatal mice. We show that gene delivery through the posterior semicircular canal is able to perfuse the entire inner ear. The easy anatomic identification of the posterior semicircular canal, as well as minimal manipulation of the temporal bone required, make this surgical approach an attractive option for inner ear gene delivery.

摘要

内耳基因治疗作为一种治疗听力损失和头晕的潜在方法具有巨大前景。内耳基因治疗成功的关键决定因素之一是找到一种递送方法,该方法能使靶向细胞类型的转导效率保持一致,同时将听力损失降至最低。在本研究中,我们描述了后半规管途径作为新生小鼠内耳基因递送的一种可行方法。我们表明,通过后半规管进行基因递送能够灌注整个内耳。后半规管易于解剖识别,且所需的颞骨操作极少,这使得这种手术方法成为内耳基因递送的一个有吸引力的选择。