Dual AAV Delivery of Otoferlin Durably Rescues Hearing in Congenitally Deaf Preclinical Models

被引：0

作者：

Whitton, Jonathon ^{[1
]}

Palermo, Adam ^{[1
]}

Becker, Lars ^{[1
]}

Burns, Joe ^{[1
]}

Pan, Ning ^{[1
]}

Schwander, Martin ^{[1
]}

Zhang, Xichun ^{[1
]}

Pan, Bifeng ^{[1
]}

Smith, Lillian ^{[1
]}

Wu, XuDong ^{[1
]}

Senapati, Arun ^{[1
]}

Gonzalez, Elizabeth Amaro ^{[1
]}

Ren, Yong ^{[1
]}

Gibson, Tyler ^{[1
]}

Abel, Theresa ^{[1
]}

Liebesny, Paul ^{[1
]}

Shi, Fuxin ^{[1
]}

Enerson, Brad ^{[1
]}

Nguyen, Ren ^{[1
]}

Druckenbrod, Noah ^{[1
]}

Alterman, Emma ^{[1
]}

Sabin, Leah ^{[2
]}

Cancelarich, Sarah ^{[2
]}

Drummond, Meghan ^{[2
]}

机构：

[1] Decibel Therapeut, Boston, MA USA

[2] Regeneron Pharmaceut, Tarrytown, NY USA

来源：

MOLECULAR THERAPY | 2020年 / 28卷 / 04期

关键词：

D O I：

暂无

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

引用

页码：2 / 3

页数：2

共 7 条

[1] A dual-AAV approach restores fast exocytosis and partially rescues auditory function in deaf otoferlin knock-out mice
Al-Moyed, Hanan
Cepeda, Andreia P.
Jung, SangYong
Moser, Tobias
Kuegler, Sebastian
Reisinger, Ellen
EMBO MOLECULAR MEDICINE, 2019, 11 (01)
[2] AAV-mediated delivery of interferon-β for the treatment of retinoblastoma in preclinical models
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Laurie, N.
Holzmacher, J.
Davidoff, A.
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[3] AAV-mediated Local Delivery of Interferon-β for the Treatment of Retinoblastoma in Preclinical Models
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Nikia Laurie
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[4] AAV-mediated Local Delivery of Interferon-β for the Treatment of Retinoblastoma in Preclinical Models
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[5] Single and Dual Vector Gene Therapy with AAV9-PHP.B Rescues Hearing in Tmc1 Mutant Mice
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[6] AAV-MEDIATED DELIVERY OF SUPPLEMENTAL TRNA RESCUES NEUROPATHY IN AMINOACYL-TRNA SYNTHETASE-ASSOCIATED MOUSE MODELS OF CHARCOT-MARIE-TOOTH DISEASE
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[7] ORAL DELIVERY OF PEPTIDES WITH DUAL MU/DELTA OPIOID RECEPTOR AGONIST ACTIVITIES ARE SUPERIOR TO ELUXADOLINE IN PRECLINICAL MODELS OF IBS-D
Mattheakis, Larry
Anandan, Sampath-Kumar
Zhao, Li
Zerangue, Noa
Zhang, Jenny
Bourne, Greg T.
Shah, Shairaz
Foulke, Matthew
Vinh Tran
Tovera, James
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GASTROENTEROLOGY, 2018, 154 (06) : S53 - S53

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