JP2023500670A - 蝸牛内耳有毛細胞プロモーター及びその使用 - Google Patents

蝸牛内耳有毛細胞プロモーター及びその使用 Download PDF

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Publication number
JP2023500670A
JP2023500670A JP2022525640A JP2022525640A JP2023500670A JP 2023500670 A JP2023500670 A JP 2023500670A JP 2022525640 A JP2022525640 A JP 2022525640A JP 2022525640 A JP2022525640 A JP 2022525640A JP 2023500670 A JP2023500670 A JP 2023500670A
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Prior art keywords
ihc
nucleic acid
acid vector
protein
polynucleotide
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Pending
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JP2022525640A
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Japanese (ja)
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JP2023500670A5 (https=
Inventor
バーンズ、ジョセフ
パレルモ、アダム
プレーゲルニッヒ、ガブリエラ
シュワンダー、マーティン
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Decibel Therapeutics Inc
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Decibel Therapeutics Inc
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Application filed by Decibel Therapeutics Inc filed Critical Decibel Therapeutics Inc
Publication of JP2023500670A publication Critical patent/JP2023500670A/ja
Publication of JP2023500670A5 publication Critical patent/JP2023500670A5/ja
Pending legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645—Adeno-associated virus
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/761—Adenovirus
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0046—Ear
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Virology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Mycology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2022525640A 2019-11-04 2020-11-04 蝸牛内耳有毛細胞プロモーター及びその使用 Pending JP2023500670A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962930503P 2019-11-04 2019-11-04
US62/930,503 2019-11-04
PCT/US2020/058782 WO2021091940A1 (en) 2019-11-04 2020-11-04 Cochlear inner hair cell promoters and uses thereof

Publications (2)

Publication Number Publication Date
JP2023500670A true JP2023500670A (ja) 2023-01-10
JP2023500670A5 JP2023500670A5 (https=) 2023-11-10

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ID=75848644

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JP2022525640A Pending JP2023500670A (ja) 2019-11-04 2020-11-04 蝸牛内耳有毛細胞プロモーター及びその使用

Country Status (5)

Country Link
US (1) US12545930B2 (https=)
EP (1) EP4055175A4 (https=)
JP (1) JP2023500670A (https=)
CN (1) CN115052989B (https=)
WO (1) WO2021091940A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN121263526A (zh) * 2023-05-23 2026-01-02 苏州星奥拓维生物技术有限公司 耳蜗毛细胞特异性启动子及其用途
CN116925239B (zh) * 2023-07-17 2024-10-18 苏州星奥拓维生物技术有限公司 双载体系统表达Otof基因的组合物和方法
CN117486995A (zh) * 2023-11-03 2024-02-02 首都医科大学附属北京胸科医院 Kncn蛋白在结核诊断试剂中的应用

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2018536420A (ja) * 2015-12-11 2018-12-13 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
WO2019138030A1 (de) * 2018-01-12 2019-07-18 Georg-August-Universität Göttingen Stiftung Öffentlichen Rechts, Universitätsmedizin Gentherapeutische behandlung von schwerhörigkeit

Family Cites Families (6)

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Publication number Priority date Publication date Assignee Title
CA2817256A1 (en) * 2010-11-12 2012-05-18 The General Hospital Corporation Polycomb-associated non-coding rnas
US20180305689A1 (en) * 2015-04-22 2018-10-25 Mina Therapeutics Limited Sarna compositions and methods of use
WO2018145111A1 (en) * 2017-02-06 2018-08-09 Children's Medical Center Corporation Materials and methods for delivering nucleic acids to cochlear and vestibular cells
CN111902539B (zh) * 2018-02-07 2025-05-16 吉尼松公司 杂合调控元件
DE102018103924A1 (de) * 2018-02-21 2019-08-22 Georg-August-Universität Göttingen Stiftung Öffentlichen Rechts, Universitätsmedizin Gentherapeutische Behandlung von Schwerhörigkeit
CN112703016B (zh) * 2018-04-27 2024-11-22 分贝治疗公司 肌球蛋白15启动子及其用途

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2018536420A (ja) * 2015-12-11 2018-12-13 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
WO2019138030A1 (de) * 2018-01-12 2019-07-18 Georg-August-Universität Göttingen Stiftung Öffentlichen Rechts, Universitätsmedizin Gentherapeutische behandlung von schwerhörigkeit

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
"Accession No.AC109138.10", DATABASE GENBANK, JPN6024038522, 1 October 2003 (2003-10-01), ISSN: 0005426824 *
"Accession No.AP001184.4", DATABASE GENBANK, JPN6024038521, 27 April 2002 (2002-04-27), ISSN: 0005426823 *

Also Published As

Publication number Publication date
CN115052989A (zh) 2022-09-13
EP4055175A1 (en) 2022-09-14
WO2021091940A1 (en) 2021-05-14
US12545930B2 (en) 2026-02-10
EP4055175A4 (en) 2023-11-29
US20220348965A1 (en) 2022-11-03
CN115052989B (zh) 2024-08-13

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