IL304674B2 - Reagents for treating Oculopharyngeal Muscular Dystrophy (OPMD) and their uses - Google Patents
Reagents for treating Oculopharyngeal Muscular Dystrophy (OPMD) and their usesInfo
- Publication number
- IL304674B2 IL304674B2 IL304674A IL30467423A IL304674B2 IL 304674 B2 IL304674 B2 IL 304674B2 IL 304674 A IL304674 A IL 304674A IL 30467423 A IL30467423 A IL 30467423A IL 304674 B2 IL304674 B2 IL 304674B2
- Authority
- IL
- Israel
- Prior art keywords
- seq
- set forth
- nucleic acid
- shmir
- effector
- Prior art date
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- C12N2740/10011—Retroviridae
- C12N2740/10041—Use of virus, viral particle or viral elements as a vector
- C12N2740/10043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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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
- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
- C12N2740/15041—Use of virus, viral particle or viral elements as a vector
- C12N2740/15043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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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
- 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
-
- 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
- C12N2800/00—Nucleic acids vectors
- C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
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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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
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- Health & Medical Sciences (AREA)
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- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
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- Veterinary Medicine (AREA)
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- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
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- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Ophthalmology & Optometry (AREA)
- Epidemiology (AREA)
- Virology (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Investigating Or Analysing Biological Materials (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662434312P | 2016-12-14 | 2016-12-14 | |
| PCT/AU2017/051385 WO2018107228A1 (en) | 2016-12-14 | 2017-12-14 | Reagents for treatment of oculopharyngeal muscular dystrophy (opmd) and use thereof |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| IL304674A IL304674A (en) | 2023-09-01 |
| IL304674B1 IL304674B1 (en) | 2024-07-01 |
| IL304674B2 true IL304674B2 (en) | 2024-11-01 |
Family
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Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL304674A IL304674B2 (en) | 2016-12-14 | 2017-12-14 | Reagents for treating Oculopharyngeal Muscular Dystrophy (OPMD) and their uses |
| IL267353A IL267353B2 (en) | 2016-12-14 | 2017-12-14 | Reagents for the treatment of (OCULOPHARYNGEAL MUSCULAR DYSTROPHY (OPMD) and their uses |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL267353A IL267353B2 (en) | 2016-12-14 | 2017-12-14 | Reagents for the treatment of (OCULOPHARYNGEAL MUSCULAR DYSTROPHY (OPMD) and their uses |
Country Status (12)
| Country | Link |
|---|---|
| US (2) | US11142765B2 (https=) |
| EP (1) | EP3555294A4 (https=) |
| JP (2) | JP7233149B2 (https=) |
| KR (2) | KR102495222B1 (https=) |
| CN (2) | CN110382698B (https=) |
| AU (2) | AU2017377661B2 (https=) |
| CA (1) | CA3047154A1 (https=) |
| IL (2) | IL304674B2 (https=) |
| MX (2) | MX2019007110A (https=) |
| RU (1) | RU2763319C2 (https=) |
| WO (1) | WO2018107228A1 (https=) |
| ZA (1) | ZA202106827B (https=) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP7233149B2 (ja) | 2016-12-14 | 2023-03-06 | ベニテック アイピー ホールディングス インコーポレーテッド | 眼咽頭型筋ジストロフィー（ｏｐｍｄ）を治療するための試薬及びその使用 |
| JP2022508835A (ja) * | 2018-10-17 | 2022-01-19 | ベニテック バイオファーマ リミテッド | 眼咽頭筋ジストロフィー（ｏｐｍｄ）を治療するための方法 |
| CN113631706A (zh) * | 2019-02-28 | 2021-11-09 | 贝尼泰克知识产权控股股份有限公司 | 用于治疗眼咽肌营养不良(opmd)的组合物和方法 |
| JP2023507501A (ja) * | 2019-12-23 | 2023-02-22 | ヴェルサメブ アーゲー | 遺伝子の発現を同時に調節するための組成物及び方法 |
| WO2026011009A1 (en) * | 2024-07-02 | 2026-01-08 | Kate Therapeutics, Inc. | Compositions and methods for muscle disorders |
Family Cites Families (10)
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| IL159756A0 (en) * | 2001-07-12 | 2004-06-20 | Univ Massachusetts | IN VIVO PRODUCTION OF SMALL INTERFERING RNAs THAT MEDIATE GENE SILENCING |
| EP1783645A1 (en) * | 2005-11-08 | 2007-05-09 | Actigenics | Methods for the identification of microRNA and their applications in research and human health |
| EP2275560A3 (en) * | 2007-01-31 | 2012-01-25 | BASF Plant Science GmbH | Trangenic plants wherein the transgene is a memeber of the REMORIN gene family or a consensus sequence thereof having enhanced yield-related traits and a method for making the same |
| DK2164967T3 (en) * | 2007-05-31 | 2015-10-19 | Univ Iowa Res Found | Reduction of off-target rna interferenstoksicitet |
| WO2010105372A1 (en) * | 2009-03-20 | 2010-09-23 | Protiva Biotherapeutics, Inc. | Compositions and methods for silencing hepatitis c virus expression |
| EP2814514B1 (en) * | 2012-02-16 | 2017-09-13 | Atyr Pharma, Inc. | Histidyl-trna synthetases for treating autoimmune and inflammatory diseases |
| WO2014077693A1 (en) * | 2012-11-16 | 2014-05-22 | Academisch Ziekenhuis Leiden H.O.D.N. Lumc | Means and methods for reducing an effect of aging in a mammalian cell |
| KR20150103280A (ko) * | 2013-01-08 | 2015-09-09 | 베니텍 바이오파마 리미티드 | 연령-관련 황반 변성 치료 |
| NZ747314A (en) * | 2016-04-14 | 2022-07-29 | Benitec Ip Holdings Inc | Reagents for treatment of oculopharyngeal muscular dystrophy (opmd) and use thereof |
| JP7233149B2 (ja) * | 2016-12-14 | 2023-03-06 | ベニテック アイピー ホールディングス インコーポレーテッド | 眼咽頭型筋ジストロフィー（ｏｐｍｄ）を治療するための試薬及びその使用 |
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2017
- 2017-12-14 JP JP2019532105A patent/JP7233149B2/ja active Active
- 2017-12-14 KR KR1020197019981A patent/KR102495222B1/ko active Active
- 2017-12-14 IL IL304674A patent/IL304674B2/en unknown
- 2017-12-14 MX MX2019007110A patent/MX2019007110A/es unknown
- 2017-12-14 EP EP17882179.9A patent/EP3555294A4/en active Pending
- 2017-12-14 AU AU2017377661A patent/AU2017377661B2/en active Active
- 2017-12-14 WO PCT/AU2017/051385 patent/WO2018107228A1/en not_active Ceased
- 2017-12-14 CA CA3047154A patent/CA3047154A1/en active Pending
- 2017-12-14 CN CN201780086338.5A patent/CN110382698B/zh active Active
- 2017-12-14 IL IL267353A patent/IL267353B2/en unknown
- 2017-12-14 US US16/469,992 patent/US11142765B2/en active Active
- 2017-12-14 RU RU2019121950A patent/RU2763319C2/ru active
- 2017-12-14 KR KR1020237003441A patent/KR102667867B1/ko active Active
- 2017-12-14 CN CN202410482087.4A patent/CN118421624A/zh active Pending
-
2019
- 2019-06-14 MX MX2024003643A patent/MX2024003643A/es unknown
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2021
- 2021-09-01 US US17/463,923 patent/US11932852B2/en active Active
- 2021-09-17 ZA ZA2021/06827A patent/ZA202106827B/en unknown
-
2023
- 2023-02-17 JP JP2023023159A patent/JP7431359B2/ja active Active
- 2023-08-28 AU AU2023222827A patent/AU2023222827B2/en active Active
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