CN113874512A - 诱导毛细胞分化的组合物和方法 - Google Patents
诱导毛细胞分化的组合物和方法 Download PDFInfo
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- CN113874512A CN113874512A CN201980087541.3A CN201980087541A CN113874512A CN 113874512 A CN113874512 A CN 113874512A CN 201980087541 A CN201980087541 A CN 201980087541A CN 113874512 A CN113874512 A CN 113874512A
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- 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
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- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4702—Regulators; Modulating activity
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- A—HUMAN NECESSITIES
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- 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
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- A61K38/00—Medicinal preparations containing peptides
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- A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
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- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/18—Growth factors; Growth regulators
- A61K38/1841—Transforming growth factor [TGF]
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- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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- A61K38/18—Growth factors; Growth regulators
- A61K38/185—Nerve growth factor [NGF]; Brain derived neurotrophic factor [BDNF]; Ciliary neurotrophic factor [CNTF]; Glial derived neurotrophic factor [GDNF]; Neurotrophins, e.g. NT-3
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- 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
- A61K48/0075—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 delivery route, e.g. oral, subcutaneous
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- 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
- A61K48/0083—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 administration regime
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- 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
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
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- 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
- A61P27/16—Otologicals
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- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/475—Growth factors; Growth regulators
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- 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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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- 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
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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- 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
- C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
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- 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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- C12N2800/00—Nucleic acids vectors
- C12N2800/40—Systems of functionally co-operating vectors
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Organic Chemistry (AREA)
- Medicinal Chemistry (AREA)
- Biotechnology (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Epidemiology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Immunology (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
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- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Virology (AREA)
- Toxicology (AREA)
- Cell Biology (AREA)
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- Neurosurgery (AREA)
- Marine Sciences & Fisheries (AREA)
- Dermatology (AREA)
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- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862756910P | 2018-11-07 | 2018-11-07 | |
| US62/756,910 | 2018-11-07 | ||
| US201962888105P | 2019-08-16 | 2019-08-16 | |
| US62/888,105 | 2019-08-16 | ||
| PCT/US2019/060324 WO2020097369A1 (en) | 2018-11-07 | 2019-11-07 | Compositions and methods of inducing differentiation of a hair cell |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CN113874512A true CN113874512A (zh) | 2021-12-31 |
Family
ID=70611182
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN201980087541.3A Pending CN113874512A (zh) | 2018-11-07 | 2019-11-07 | 诱导毛细胞分化的组合物和方法 |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US20220395582A1 (https=) |
| EP (1) | EP3880827A4 (https=) |
| JP (1) | JP2022512921A (https=) |
| CN (1) | CN113874512A (https=) |
| AU (1) | AU2019376084A1 (https=) |
| CA (1) | CA3119059A1 (https=) |
| WO (1) | WO2020097369A1 (https=) |
Families Citing this family (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2020160371A1 (en) | 2019-02-01 | 2020-08-06 | The University Of Hong Kong | Innervated organoid compositions and methods of making same |
| WO2020243613A1 (en) | 2019-05-31 | 2020-12-03 | Children's Hospital Medical Center | Methods of generating and expanding hematopoietic stem cells |
| WO2020243633A1 (en) | 2019-05-31 | 2020-12-03 | Children's Hospital Medical Center | Shaped organoid compositions and methods of making same |
| KR20230061333A (ko) * | 2020-06-23 | 2023-05-08 | 칠드런즈 호스피탈 메디칼 센터 | 인슐린 저항성 모델 |
| JP2024517843A (ja) * | 2021-05-05 | 2024-04-23 | デシベル セラピューティクス インコーポレイテッド | ステレオシリン二重ベクター系を使用して感音性難聴を治療するための組成物及び方法 |
| JP2025503508A (ja) * | 2021-12-23 | 2025-02-04 | ユニバーシティ オブ ロチェスター | 内耳への薬剤の送達のための組成物及び方法 |
| WO2025094056A1 (en) * | 2023-10-31 | 2025-05-08 | Children's Medical Research Institute | Gene therapy constructs and methods of use therefor |
Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN1315998A (zh) * | 1998-07-14 | 2001-10-03 | 科里克萨有限公司 | 用于治疗和诊断前列腺癌的组合物和方法 |
| US20040166091A1 (en) * | 2003-02-24 | 2004-08-26 | Genvec, Inc. | Materials and methods for treating disorders of the ear |
| WO2017100791A1 (en) * | 2015-12-11 | 2017-06-15 | Massachusetts Eye And Ear Infirmary | Materials and methods for delivering nucleic acids to cochlear and vestibular cells |
Family Cites Families (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20050281786A1 (en) * | 2004-06-18 | 2005-12-22 | David Poulsen | AAV mediated gene delivery to cochlear cells |
| CA2801535C (en) * | 2010-06-04 | 2017-05-30 | Hough Ear Institute | Composition and method for inner ear sensory hair cell regeneration or replacement |
| KR20140047141A (ko) * | 2011-08-03 | 2014-04-21 | 쿠아크 파마수티칼스 인코퍼레이티드 | 청력 및 균형 장애를 치료하기 위한 이중-가닥 올리고뉴클레오티드 화합물들 |
| CA2909733C (en) * | 2013-04-18 | 2023-10-17 | Fondazione Telethon | Effective delivery of large genes by dual aav vectors |
| JP2015534818A (ja) * | 2013-11-04 | 2015-12-07 | ジェネシス リサーチ インスティチュート | 聴覚を治療するための組成物および方法 |
| WO2017108931A1 (en) * | 2015-12-22 | 2017-06-29 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Improved hybrid dual recombinant aav vector systems for gene therapy |
| WO2017136764A1 (en) * | 2016-02-05 | 2017-08-10 | The General Hospital Corporation | Hybrid system for efficient gene delivery to cells of the inner ear |
| US20190142969A1 (en) * | 2016-04-26 | 2019-05-16 | Massachusetts Eye And Ear Infirmary | ISL1-Based Gene Therapy to Treat Hearing Loss |
| JP2019530737A (ja) * | 2016-08-23 | 2019-10-24 | アコーオス インコーポレイテッド | ヒト対象において非加齢性聴力障害を治療するための組成物および方法 |
| CA3115810A1 (en) * | 2018-10-11 | 2020-04-16 | Decibel Therapeutics, Inc. | Aav1 vectors and uses thereof for treatment of otic indications |
| CN110437317B (zh) * | 2019-01-30 | 2023-05-02 | 上海科技大学 | 具有变异衣壳蛋白的腺相关病毒及其用途 |
-
2019
- 2019-11-07 CA CA3119059A patent/CA3119059A1/en active Pending
- 2019-11-07 EP EP19882633.1A patent/EP3880827A4/en not_active Withdrawn
- 2019-11-07 JP JP2021524058A patent/JP2022512921A/ja active Pending
- 2019-11-07 US US17/291,906 patent/US20220395582A1/en not_active Abandoned
- 2019-11-07 CN CN201980087541.3A patent/CN113874512A/zh active Pending
- 2019-11-07 AU AU2019376084A patent/AU2019376084A1/en not_active Abandoned
- 2019-11-07 WO PCT/US2019/060324 patent/WO2020097369A1/en not_active Ceased
Patent Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN1315998A (zh) * | 1998-07-14 | 2001-10-03 | 科里克萨有限公司 | 用于治疗和诊断前列腺癌的组合物和方法 |
| US20040166091A1 (en) * | 2003-02-24 | 2004-08-26 | Genvec, Inc. | Materials and methods for treating disorders of the ear |
| WO2017100791A1 (en) * | 2015-12-11 | 2017-06-15 | Massachusetts Eye And Ear Infirmary | Materials and methods for delivering nucleic acids to cochlear and vestibular cells |
Non-Patent Citations (3)
| Title |
|---|
| BRADLEY J. WALTERS等: "In Vivo Interplay between p27Kip1 , GATA3, ATOH1, and POU4F3 Converts Non-sensory Cells to Hair Cells in Adult Mice", CELL REPORTS, vol. 19, no. 2, pages 307 - 320, XP055950275, DOI: 10.1016/j.celrep.2017.03.044 * |
| FEI ZHENG等: "Cochlear hair cell regeneration after noise-induced hearing loss: Does regeneration follow development?", HEARING RESEARCH, vol. 349, pages 182 - 196, XP085025663, DOI: 10.1016/j.heares.2016.12.011 * |
| MARI´A BEATRIZ DURAN ALONSO等: "Transcription factor induced conversion of human fibroblasts towards the hair cell lineage", PLOS ONE, vol. 13, no. 7, 6 July 2018 (2018-07-06), pages 1 - 16 * |
Also Published As
| Publication number | Publication date |
|---|---|
| WO2020097369A8 (en) | 2021-04-29 |
| AU2019376084A1 (en) | 2021-05-20 |
| JP2022512921A (ja) | 2022-02-07 |
| EP3880827A1 (en) | 2021-09-22 |
| EP3880827A4 (en) | 2023-04-26 |
| CA3119059A1 (en) | 2020-05-14 |
| WO2020097369A1 (en) | 2020-05-14 |
| US20220395582A1 (en) | 2022-12-15 |
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