Gene therapy for hereditary deafness

被引：3

作者：

Smith, Carson ^{[1
]}

Zafeer, Mohammad Faraz ^{[2
]}

Tekin, Mustafa ^{[1
,2
]}

机构：

[1] Univ Miami, Miller Sch Med, John T Macdonald Fdn Dept Human Genet, Miami, FL 33136 USA

[2] Univ Miami, Miller Sch Med, John P Hussman Inst Human Genom, Miami, FL 33136 USA

来源：

NATURE MEDICINE | 2024年 / 30卷 / 07期

关键词：

HEARING-LOSS; OTOFERLIN;

D O I：

10.1038/s41591-024-03004-8

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

071010 ; 081704 ;

摘要：

Bilateral administration of human OTOF gene therapy appears safe, and preliminary data suggest that it could effectively treat a rare form of hereditary hearing loss by enhancing speech perception, sound source localization and overall auditory experience.

引用

页码：1828 / 1829

页数：2

共 11 条

[1] Dual AAV-mediated gene therapy restores hearing in a DFNB9 mouse model [J].

Akil, Omar ;

Dyka, Frank ;

Calvet, Charlotte ;

Emptoz, Alice ;

Lahlou, Ghizlene ;

Nouaille, Sylvie ;

de Monvel, Jacques Boutet ;

Hardelin, Jean-Pierre ;

Hauswirth, William W. ;

Avan, Paul ;

Petit, Christine ;

Safieddine, Saaid ;

Lustig, Lawrence R. .

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2019, 116 (10) :4496-4501

[2] A dual-AAV approach restores fast exocytosis and partially rescues auditory function in deaf otoferlin knock-out mice [J].

Al-Moyed, Hanan ;

Cepeda, Andreia P. ;

Jung, SangYong ;

Moser, Tobias ;

Kuegler, Sebastian ;

Reisinger, Ellen .

EMBO MOLECULAR MEDICINE, 2019, 11 (01)

[3] The natural history, clinical outcomes, and genotype-phenotype relationship of otoferlin-related hearing loss: a systematic, quantitative literature review [J].

Ford, Charles L. ;

Riggs, William J. ;

Quigley, Tera ;

Keifer Jr, Orion P. ;

Whitton, Jonathon P. ;

Valayannopoulos, Vassili .

HUMAN GENETICS, 2023, 142 (10) :1429-1449

[4] Gene Therapy for Inherited Hearing Loss: Updates and Remaining Challenges [J].

Hahn, Roni ;

Avraham, Karen B. .

AUDIOLOGY RESEARCH, 2023, 13 (06) :952-966

[5]

Lv J, 2024, LANCET, V403, P2317, DOI 10.1016/S0140-6736(23)02874-X

[6] Hearing requires otoferlin-dependent efficient replenishment of synaptic vesicles in hair cells [J].

Pangrsic, Tina ;

Lasarow, Livia ;

Reuter, Kirsten ;

Takago, Hideki ;

Schwander, Martin ;

Riedel, Dietmar ;

Frank, Thomas ;

Tarantino, Lisa M. ;

Bailey, Janice S. ;

Strenzke, Nicola ;

Brose, Nils ;

Mueller, Ulrich ;

Reisinger, Ellen ;

Moser, Tobias .

NATURE NEUROSCIENCE, 2010, 13 (07) :869-U116

[7] Deafness: from genetic architecture to gene therapy [J].

Petit, Christine ;

Bonnet, Crystel ;

Safieddine, Saaid .

NATURE REVIEWS GENETICS, 2023, 24 (10) :665-686

[8] AAV-Mediated Gene Therapy Restores Hearing in Patients with DFNB9 Deafness [J].

Qi, Jieyu ;

Tan, Fangzhi ;

Zhang, Liyan ;

Lu, Ling ;

Zhang, Shanzhong ;

Zhai, Yabo ;

Lu, Yicheng ;

Qian, Xiaoyun ;

Dong, Wenxiu ;

Zhou, Yinyi ;

Zhang, Ziyu ;

Yang, Xuehan ;

Jiang, Lulu ;

Yu, Chaorong ;

Liu, Jiancheng ;

Chen, Tian ;

Wu, Lianqiu ;

Tan, Chang ;

Sun, Sijie ;

Song, Huaien ;

Shu, Yilai ;

Xu, Lei ;

Gao, Xia ;

Li, Huawei ;

Chai, Renjie .

ADVANCED SCIENCE, 2024, 11 (11)

[9] Genome sequencing identifies coding and non-coding variants for non-syndromic hearing loss [J].

Ramzan, Memoona ;

Duman, Duygu ;

Hendricks, LeShon Chere Peart ;

Guo, Shengru ;

Mutlu, Ahmet ;

Kalcioglu, Mahmut Tayyar ;

Seyhan, Serhat ;

Carranza, Claudia ;

Bonyadi, Murtaza ;

Mahdieh, Nejat ;

Yildirim-Baylan, Muzeyyen ;

Figueroa-Ildefonso, Erick ;

Alper, Ozgul ;

Atik, Tahir ;

Ayral, Abdurrahman ;

Bozan, Nazim ;

Balta, Burhan ;

Rivas, Christian ;

Manzoli, Gabrielle N. ;

Huesca-Hernandez, Fabiola ;

Kuchay, Raja A. H. ;

Durgut, Merve ;

Bademci, Guney ;

Tekin, Mustafa .

JOURNAL OF HUMAN GENETICS, 2023, 68 (10) :657-669

[10] Bilateral gene therapy in children with autosomal recessive deafness 9: single-arm trial results [J].

Wang, Hui ;

Chen, Yuxin ;

Lv, Jun ;

Cheng, Xiaoting ;

Cao, Qi ;

Wang, Daqi ;

Zhang, Longlong ;

Zhu, Biyun ;

Shen, Min ;

Xu, Chunxin ;

Xun, Mengzhao ;

Wang, Zijing ;

Tang, Honghai ;

Hu, Shaowei ;

Cui, Chong ;

Jiang, Luoying ;

Yin, Yanbo ;

Guo, Luo ;

Zhou, Yi ;

Han, Lei ;

Gao, Ziwen ;

Zhang, Jiajia ;

Yu, Sha ;

Gao, Kaiyu ;

Wang, Jinghan ;

Chen, Bing ;

Wang, Wuqing ;

Chen, Zheng-Yi ;

Li, Huawei ;

Shu, Yilai .

NATURE MEDICINE, 2024, 30 (07) :1898-1904

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