Hearing of Otof-deficient mice restored by trans-splicing of N- and C-terminal otoferlin-Reference-Cited by-同舟云学术

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Hearing of Otof-deficient mice restored by trans-splicing of N- and C-terminal otoferlin

Published:2022-11-16 Issue:2 Volume:142 Page:289-304
ISSN:0340-6717
Container-title:Human Genetics
language:en
Short-container-title:Hum Genet

Author:

Tang Honghai,Wang Hui,Wang Shengyi,Hu Shao Wei,Lv Jun,Xun Mengzhao,Gao Kaiyu,Wang Fang,Chen Yuxin,Wang Daqi,Wang Wuqing,Li Huawei,Shu Yilai^ORCID

Funder

the National Key Research and Development Program of China

the National Natural Science Foundation of China

the Clinical Research Plan of SHDC

the Science and Technology Commission of Shanghai Municipality

the Special Project for Clinical Research in Health Industry of Shanghai Municipal Health Commission

the Excellent Doctors-Excellent Clinical Researchers Program of Eye & ENT Hospital of Fudan University

the “Shuguang Program” supported by the Shanghai Education Development Foundation and the Shanghai Municipal Education Commission

Publisher

Springer Science and Business Media LLC

Subject

Genetics (clinical),Genetics

Link

https://link.springer.com/content/pdf/10.1007/s00439-022-02504-2.pdf

Reference66 articles.

1. Adato A, Raskin L, Petit C, Bonne-Tamir B (2000) Deafness heterogeneity in a Druze isolate from the Middle East: novel OTOF and PDS mutations, low prevalence of GJB2 35delG mutation and indication for a new DFNB locus. Eur J Hum Genet 8:437–442. https://doi.org/10.1038/sj.ejhg.5200489

2. Akil O, Dyka F, Calvet C, Emptoz A, Lahlou G, Nouaille S, de Monvel JB, Hardelin JP, Hauswirth WW, Avan P, Petit C, Safieddine S, Lustig LR (2019) Dual AAV-mediated gene therapy restores hearing in a DFNB9 mouse model. Proc Natl Acad Sci USA 116:4496–4501. https://doi.org/10.1073/pnas.1817537116

3. Al-Moyed H, Cepeda AP, Jung SY, Moser T, Kugler S, Reisinger E (2019) A dual-AAV approach restores fast exocytosis and partially rescues auditory function in deaf otoferlin knock-out mice. EMBO Mol Med 11:1–13

4. Anzalone AV, Koblan LW, Liu DR (2020) Genome editing with CRISPR-Cas nucleases, base editors, transposases and prime editors. Nat Biotechnol 38:824–844. https://doi.org/10.1038/s41587-020-0561-9

5. Anzalone AV, Randolph PB, Davis JR, Sousa AA, Koblan LW, Levy JM, Chen PJ, Wilson C, Newby GA, Raguram A, Liu DR (2019) Search-and-replace genome editing without double-strand breaks or donor DNA. Nature 576:149–157. https://doi.org/10.1038/s41586-019-1711-4

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1. Hearing restoration by gene replacement therapy for a multisite-expressed gene in a mouse model of human DFNB111 deafness;The American Journal of Human Genetics;2024-09

2. A base editor for the long-term restoration of auditory function in mice with recessive profound deafness;Nature Biomedical Engineering;2024-08-12

3. Gene therapy advancements for the treatment of acquired and hereditary hearing loss;Frontiers in Audiology and Otology;2024-07-17

4. Breaking the silence: gene therapy offers hope for OTOF-mediated hearing loss, editorial;Annals of Medicine & Surgery;2024-07-08

5. Recovery kinetics of dual AAV-mediated human otoferlin expression;Frontiers in Molecular Neuroscience;2024-06-17

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