CN110709060A - 用于治疗听力丧失的基因治疗构建体和方法 - Google Patents

用于治疗听力丧失的基因治疗构建体和方法 Download PDF

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Publication number
CN110709060A
CN110709060A CN201880028001.3A CN201880028001A CN110709060A CN 110709060 A CN110709060 A CN 110709060A CN 201880028001 A CN201880028001 A CN 201880028001A CN 110709060 A CN110709060 A CN 110709060A
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promoter
nucleic acid
tmprss3
hearing loss
seq
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Chinese (zh)
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H·施特克尔
X·Z·刘
陈正一
C·J·阿亚拉
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Rescue Hearing Co ltd
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Rescue Hearing Co ltd
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CN201880028001.3A 2017-03-17 2018-03-16 用于治疗听力丧失的基因治疗构建体和方法 Pending CN110709060A (zh)

Applications Claiming Priority (5)

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US201762472790P 2017-03-17 2017-03-17
US62/472,790 2017-03-17
US201762531522P 2017-07-12 2017-07-12
US62/531,522 2017-07-12
PCT/US2018/022873 WO2018170402A1 (en) 2017-03-17 2018-03-16 Gene therapy constructs and methods for treatment of hearing loss

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CN110709060A true CN110709060A (zh) 2020-01-17

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US (2) US20200248203A1 (enExample)
EP (1) EP3595634B1 (enExample)
JP (1) JP2020509786A (enExample)
KR (1) KR102752457B1 (enExample)
CN (1) CN110709060A (enExample)
BR (1) BR112019019367A2 (enExample)
CA (1) CA3054941A1 (enExample)
ES (1) ES2982270T3 (enExample)
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Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113584082A (zh) * 2021-06-22 2021-11-02 复旦大学附属眼耳鼻喉科医院 CRISPR/Cas9基因编辑系统及其在制备治疗遗传性感音神经性聋的药物中的应用
CN116096391A (zh) * 2020-02-05 2023-05-09 援救听觉股份有限公司 用于治疗听力损失的基因疗法系统和相关方法

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12391936B2 (en) 2017-03-17 2025-08-19 Rescue Hearing, Inc. Gene therapy systems and related methods for treatment of hearing loss
CN115820654A (zh) * 2019-08-30 2023-03-21 深圳华大基因股份有限公司 Loxhd1基因突变体及其应用
EP4008788A1 (en) * 2020-12-03 2022-06-08 Medizinische Hochschule Hannover Lentiviral vector technology for inner ear gene therapy
CN113215248B (zh) * 2021-06-25 2022-04-22 中国人民解放军空军军医大学 一种感音神经性耳聋相关的myo15a基因突变检测试剂盒
WO2024259309A1 (en) * 2023-06-15 2024-12-19 Regeneron Pharmaceuticals, Inc. Gene therapy for hearing disorders

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2004099779A1 (en) * 2003-05-05 2004-11-18 Bayer Healthcare Ag Diagnostics and therapeutics for diseases associated with human transmembrane serine protease 3 (tmprss3)
US20130095071A1 (en) * 2009-12-21 2013-04-18 Audigen Inc. Method of treating or preventing hearing loss
US20150329875A1 (en) * 2014-05-13 2015-11-19 Sangamo Biosciences, Inc. Methods and compositions for prevention or treatment of a disease

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1280923A2 (en) * 2000-04-28 2003-02-05 Millennium Pharmaceuticals, Inc. 14094, a human trypsin family member and uses thereof
ZA200800970B (en) * 2005-08-15 2009-10-28 Genentech Inc Gene disruptions, compositions and methods relating thereto
US20150050354A1 (en) * 2012-04-02 2015-02-19 Moderna Therapeutics, Inc. Modified polynucleotides for the treatment of otic diseases and conditions
EP2877213B1 (en) 2012-07-25 2020-12-02 The Broad Institute, Inc. Inducible dna binding proteins and genome perturbation tools and applications thereof
US20160237455A1 (en) 2013-09-27 2016-08-18 Editas Medicine, Inc. Crispr-related methods and compositions
JP6552511B2 (ja) 2013-10-11 2019-07-31 マサチューセッツ アイ アンド イヤー インファーマリー 祖先ウイルス配列を予測する方法およびその使用
WO2016069910A1 (en) 2014-10-29 2016-05-06 Massachusetts Eye And Ear Infirmary Methods for efficient delivery of therapeutic molecules in vitro and in vivo
JP6990182B2 (ja) * 2015-12-11 2022-02-15 マサチューセッツ アイ アンド イヤー インファーマリー 蝸牛および前庭細胞に核酸を送達するための材料および方法

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2004099779A1 (en) * 2003-05-05 2004-11-18 Bayer Healthcare Ag Diagnostics and therapeutics for diseases associated with human transmembrane serine protease 3 (tmprss3)
US20130095071A1 (en) * 2009-12-21 2013-04-18 Audigen Inc. Method of treating or preventing hearing loss
US20150329875A1 (en) * 2014-05-13 2015-11-19 Sangamo Biosciences, Inc. Methods and compositions for prevention or treatment of a disease

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
JUYONG CHUNG等: "A novel mutation of TMPRSS3 related to milder auditory phenotype in Korean postlingual deafness：a possible future implication for a personalized auditory rehabilitation", 《J MOL MED》, vol. 93, pages 651 - 663 *
NICOLAS GRILLET等: "Mutations in LOXHD1, an Evolutionarily Conserved Stereociliary Protein, Disrupt Hair Cell Function in Mice and Cause Progressive Hearing Loss in Humans", 《THE AMERICAN JOURNAL OF HUMAN GENETICS》 *
NICOLAS GRILLET等: "Mutations in LOXHD1, an Evolutionarily Conserved Stereociliary Protein, Disrupt Hair Cell Function in Mice and Cause Progressive Hearing Loss in Humans", 《THE AMERICAN JOURNAL OF HUMAN GENETICS》, vol. 85, 11 September 2009 (2009-09-11), pages 328 - 337, XP055732361, DOI: 10.1016/j.ajhg.2009.07.017 *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN116096391A (zh) * 2020-02-05 2023-05-09 援救听觉股份有限公司 用于治疗听力损失的基因疗法系统和相关方法
CN113584082A (zh) * 2021-06-22 2021-11-02 复旦大学附属眼耳鼻喉科医院 CRISPR/Cas9基因编辑系统及其在制备治疗遗传性感音神经性聋的药物中的应用

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US20200114023A1 (en) 2020-04-16
US20200248203A1 (en) 2020-08-06
KR20190131052A (ko) 2019-11-25
CA3054941A1 (en) 2018-09-20
KR102752457B1 (ko) 2025-01-09
IL268912B1 (en) 2024-07-01
EP3595634A4 (en) 2020-11-04
JP2020509786A (ja) 2020-04-02
WO2018170402A1 (en) 2018-09-20
IL268912A (en) 2019-10-31
EP3595634A1 (en) 2020-01-22
ES2982270T3 (es) 2024-10-15
IL268912B2 (en) 2024-11-01
EP3595634B1 (en) 2024-03-27

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