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1
Cochlear gene therapy.耳蜗基因治疗。
Curr Opin Neurol. 2012 Feb;25(1):57-60. doi: 10.1097/WCO.0b013e32834f038c.
2
Recent advances in cochlear hair cell regeneration-A promising opportunity for the treatment of age-related hearing loss.耳蜗毛细胞再生的最新进展——治疗与年龄相关的听力损失的有希望的机会。
Ageing Res Rev. 2017 Jul;36:149-155. doi: 10.1016/j.arr.2017.04.002. Epub 2017 Apr 13.
3
Virally Mediated Overexpression of Glial-Derived Neurotrophic Factor Elicits Age- and Dose-Dependent Neuronal Toxicity and Hearing Loss.病毒介导的胶质源性神经营养因子过表达引起年龄和剂量依赖性神经元毒性和听力损失。
Hum Gene Ther. 2019 Jan;30(1):88-105. doi: 10.1089/hum.2018.028. Epub 2018 Sep 5.
4
Cochlear Gene Therapy.耳蜗基因治疗。
Cold Spring Harb Perspect Med. 2019 Sep 3;9(9):a033191. doi: 10.1101/cshperspect.a033191.
5
Regeneration of stereocilia of hair cells by forced Atoh1 expression in the adult mammalian cochlea.成年哺乳动物耳蜗中强制表达 Atoh1 可使毛细胞的静纤毛再生。
PLoS One. 2012;7(9):e46355. doi: 10.1371/journal.pone.0046355. Epub 2012 Sep 27.
6
Manipulating cell fate in the cochlea: a feasible therapy for hearing loss.操控耳蜗中的细胞命运:一种治疗听力损失的可行疗法。
Trends Neurosci. 2015 Mar;38(3):139-44. doi: 10.1016/j.tins.2014.12.004. Epub 2015 Jan 12.
7
Establishment of a model of cochlear lesions in rats to study potential gene therapy for sensorineural hearing loss.建立大鼠耳蜗损伤模型以研究感音神经性听力损失的潜在基因治疗方法。
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8
New molecular therapies for the treatment of hearing loss.用于治疗听力损失的新型分子疗法。
Pharmacol Ther. 2019 Aug;200:190-209. doi: 10.1016/j.pharmthera.2019.05.003. Epub 2019 May 8.
9
Cochlear pathology, sensory cell death and regeneration.耳蜗病理学、感觉细胞死亡与再生。
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Adeno-associated virus-mediated gene delivery into the scala media of the normal and deafened adult mouse ear.腺相关病毒介导的基因递送至正常和聋鼠内耳中阶的研究。
Gene Ther. 2011 Jun;18(6):569-78. doi: 10.1038/gt.2010.175. Epub 2011 Jan 6.

引用本文的文献

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Improving Control of Gene Therapy-Based Neurotrophin Delivery for Inner Ear Applications.改善基于基因疗法的神经营养因子内耳递送的控制
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2
AudioGene: refining the natural history of KCNQ4, GSDME, WFS1, and COCH-associated hearing loss.音频基因:KCNQ4、GSDME、WFS1 和 COCH 相关听力损失的自然病史研究。
Hum Genet. 2022 Apr;141(3-4):877-887. doi: 10.1007/s00439-021-02424-7. Epub 2022 Jan 17.
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Development of in-house genetic screening for pediatric hearing loss.用于小儿听力损失的内部基因筛查的开发。
Laryngoscope Investig Otolaryngol. 2020 May 20;5(3):497-505. doi: 10.1002/lio2.379. eCollection 2020 Jun.
4
Neurotrophin gene therapy to promote survival of spiral ganglion neurons after deafness.神经营养因子基因治疗促进耳聋后螺旋神经节神经元的存活。
Hear Res. 2020 Sep 1;394:107955. doi: 10.1016/j.heares.2020.107955. Epub 2020 Apr 5.
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AAV-Mediated Neurotrophin Gene Therapy Promotes Improved Survival of Cochlear Spiral Ganglion Neurons in Neonatally Deafened Cats: Comparison of AAV2-hBDNF and AAV5-hGDNF.AAV 介导的神经营养因子基因治疗促进新生聋猫耳蜗螺旋神经节神经元存活的改善:AAV2-hBDNF 和 AAV5-hGDNF 的比较。
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Transduction of Adeno-Associated Virus Vectors Targeting Hair Cells and Supporting Cells in the Neonatal Mouse Cochlea.靶向新生小鼠耳蜗毛细胞和支持细胞的腺相关病毒载体转导
Front Cell Neurosci. 2019 Jan 24;13:8. doi: 10.3389/fncel.2019.00008. eCollection 2019.
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Neural crest contributions to the ear: Implications for congenital hearing disorders.神经嵴对耳朵的贡献:对先天性听力障碍的影响。
Hear Res. 2019 May;376:22-32. doi: 10.1016/j.heares.2018.11.005. Epub 2018 Nov 14.
8
Virally Mediated Overexpression of Glial-Derived Neurotrophic Factor Elicits Age- and Dose-Dependent Neuronal Toxicity and Hearing Loss.病毒介导的胶质源性神经营养因子过表达引起年龄和剂量依赖性神经元毒性和听力损失。
Hum Gene Ther. 2019 Jan;30(1):88-105. doi: 10.1089/hum.2018.028. Epub 2018 Sep 5.
9
Outlook and future of inner ear therapy.内耳治疗的前景展望。
Hear Res. 2018 Oct;368:127-135. doi: 10.1016/j.heares.2018.05.009. Epub 2018 May 17.
10
Ototoxicity: A Challenge in Diagnosis and Treatment.耳毒性:诊断与治疗中的一项挑战。
J Audiol Otol. 2018 Apr;22(2):59-68. doi: 10.7874/jao.2017.00360. Epub 2018 Feb 26.

本文引用的文献

1
Towards gene therapy for deafness.迈向耳聋的基因治疗。
J Cell Physiol. 2011 Oct;226(10):2494-9. doi: 10.1002/jcp.22617.
2
Experimental autoimmune hearing loss is exacerbated in IL-10-deficient mice and reversed by IL-10 gene transfer.实验性自身免疫性听力损失在白细胞介素-10 缺陷型小鼠中加重,并可通过白细胞介素-10 基因转移逆转。
Gene Ther. 2012 Feb;19(2):228-35. doi: 10.1038/gt.2011.88. Epub 2011 Jun 23.
3
Efficient cochlear gene transfection in guinea-pigs with adeno-associated viral vectors by partial digestion of round window membrane.腺相关病毒载体经圆窗膜部分消化促进豚鼠耳蜗内高效基因转染。
Gene Ther. 2012 Mar;19(3):255-63. doi: 10.1038/gt.2011.91. Epub 2011 Jun 23.
4
The effect of deafness duration on neurotrophin gene therapy for spiral ganglion neuron protection.耳聋时间对螺旋神经节神经元保护的神经营养因子基因治疗的影响。
Hear Res. 2011 Aug;278(1-2):69-76. doi: 10.1016/j.heares.2011.04.010. Epub 2011 May 1.
5
Nuclear entry of hyperbranched polylysine nanoparticles into cochlear cells.超支化多聚赖氨酸纳米颗粒进入耳蜗细胞的核内。
Int J Nanomedicine. 2011;6:535-46. doi: 10.2147/IJN.S16973. Epub 2011 Mar 14.
6
Adeno-associated virus-mediated gene delivery into the scala media of the normal and deafened adult mouse ear.腺相关病毒介导的基因递送至正常和聋鼠内耳中阶的研究。
Gene Ther. 2011 Jun;18(6):569-78. doi: 10.1038/gt.2010.175. Epub 2011 Jan 6.
7
Adeno-associated viral vector-mediated expression of NT4-ADNF-9 fusion gene protects against aminoglycoside-induced auditory hair cell loss in vitro.腺相关病毒载体介导的NT4-ADNF-9融合基因表达在体外可保护免受氨基糖苷类药物诱导的听觉毛细胞损失。
Acta Otolaryngol. 2011 Feb;131(2):136-41. doi: 10.3109/00016489.2010.518974. Epub 2010 Nov 9.
8
Hearing impairment: a panoply of genes and functions.听力障碍:基因与功能的全景图。
Neuron. 2010 Oct 21;68(2):293-308. doi: 10.1016/j.neuron.2010.10.011.
9
Profound deafness in childhood.儿童期重度耳聋。
N Engl J Med. 2010 Oct 7;363(15):1438-50. doi: 10.1056/NEJMra0911225.
10
Hair cell fate decisions in cochlear development and regeneration.耳蜗发育和再生中的毛细胞命运决定。
Hear Res. 2010 Jul;266(1-2):18-25. doi: 10.1016/j.heares.2010.04.012. Epub 2010 May 5.

耳蜗基因治疗。

Cochlear gene therapy.

机构信息

Department of Otolaryngology-Head & Neck Surgery, University of California, San Francisco, San Francisco, California 94115, USA.

出版信息

Curr Opin Neurol. 2012 Feb;25(1):57-60. doi: 10.1097/WCO.0b013e32834f038c.

DOI:10.1097/WCO.0b013e32834f038c
PMID:22157110
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC3755754/
Abstract

PURPOSE OF REVIEW

This review highlights recent advances in cochlear gene therapy over the past several years. Cochlear gene therapy has undergone tremendous advances over the past decade. Beginning with some groundbreaking work in 2005 documenting hair cell regeneration using virally mediated delivery of the mouse atonal 1 gene, gene therapy is now being explored as a possible treatment for a variety of causes of hearing loss.

RECENT FINDINGS

Recent advances in cochlear gene therapy include improved methods of gene delivery with a better delineation of viral vectors that are suitable for this purpose, additional improvements in hair cell regeneration, and directed research toward autoimmune hearing loss, ototoxicity, spiral ganglion survival, and genetic forms of hearing loss.

SUMMARY

If successful, cochlear gene therapy will dramatically alter our ability to treat a variety of forms of acquired and genetic hearing loss.

摘要

目的综述

本篇综述重点介绍了过去几年中耳蜗基因治疗的最新进展。过去十年中,耳蜗基因治疗取得了巨大进展。从 2005 年开创性地使用病毒介导的小鼠 atonal 1 基因转导实现毛细胞再生开始,基因治疗现在正被探索作为治疗各种原因听力损失的一种可能方法。

最近的发现

耳蜗基因治疗的最新进展包括改进了基因传递方法,更好地确定了适合该目的的病毒载体,毛细胞再生方面的进一步改进,以及针对自身免疫性听力损失、耳毒性、螺旋神经节存活和遗传性听力损失的定向研究。

总结

如果成功,耳蜗基因治疗将极大地改变我们治疗各种获得性和遗传性听力损失的能力。