JP5931720B2 - Dmdファミリーに対する天然アンチセンス転写物の抑制によるジストロフィンファミリー関連疾患の治療 - Google Patents
Dmdファミリーに対する天然アンチセンス転写物の抑制によるジストロフィンファミリー関連疾患の治療 Download PDFInfo
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- JP5931720B2 JP5931720B2 JP2012510006A JP2012510006A JP5931720B2 JP 5931720 B2 JP5931720 B2 JP 5931720B2 JP 2012510006 A JP2012510006 A JP 2012510006A JP 2012510006 A JP2012510006 A JP 2012510006A JP 5931720 B2 JP5931720 B2 JP 5931720B2
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
- A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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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
- A61P27/02—Ophthalmic agents
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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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
- A61P9/04—Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
- A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
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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
- C12N15/11—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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- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
- C12N2310/113—Antisense targeting other non-coding nucleic acids, e.g. antagomirs
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- Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
- Y10—TECHNICAL SUBJECTS COVERED BY FORMER USPC
- Y10T—TECHNICAL SUBJECTS COVERED BY FORMER US CLASSIFICATION
- Y10T436/00—Chemistry: analytical and immunological testing
- Y10T436/14—Heterocyclic carbon compound [i.e., O, S, N, Se, Te, as only ring hetero atom]
- Y10T436/142222—Hetero-O [e.g., ascorbic acid, etc.]
- Y10T436/143333—Saccharide [e.g., DNA, etc.]
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Biochemistry (AREA)
- Epidemiology (AREA)
- Cardiology (AREA)
- Neurology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Neurosurgery (AREA)
- Heart & Thoracic Surgery (AREA)
- Hospice & Palliative Care (AREA)
- Ophthalmology & Optometry (AREA)
- Psychiatry (AREA)
- Urology & Nephrology (AREA)
- Vascular Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US17659409P | 2009-05-08 | 2009-05-08 | |
| US61/176,594 | 2009-05-08 | ||
| US31735010P | 2010-03-25 | 2010-03-25 | |
| US61/317,350 | 2010-03-25 | ||
| PCT/US2010/034026 WO2010129861A2 (en) | 2009-05-08 | 2010-05-07 | Treatment of dystrophin family related diseases by inhibition of natural antisense transcript to dmd family |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2012525852A JP2012525852A (ja) | 2012-10-25 |
| JP2012525852A5 JP2012525852A5 (https=) | 2013-06-20 |
| JP5931720B2 true JP5931720B2 (ja) | 2016-06-08 |
Family
ID=43050898
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2012510006A Expired - Fee Related JP5931720B2 (ja) | 2009-05-08 | 2010-05-07 | Dmdファミリーに対する天然アンチセンス転写物の抑制によるジストロフィンファミリー関連疾患の治療 |
Country Status (8)
| Country | Link |
|---|---|
| US (2) | US9012139B2 (https=) |
| EP (1) | EP2427554B1 (https=) |
| JP (1) | JP5931720B2 (https=) |
| KR (1) | KR101742334B1 (https=) |
| CN (1) | CN102459597B (https=) |
| CA (2) | CA2761248C (https=) |
| ES (1) | ES2618572T3 (https=) |
| WO (1) | WO2010129861A2 (https=) |
Families Citing this family (40)
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| US9920317B2 (en) | 2010-11-12 | 2018-03-20 | The General Hospital Corporation | Polycomb-associated non-coding RNAs |
| EP3260540A1 (en) | 2010-11-12 | 2017-12-27 | The General Hospital Corporation | Polycomb-associated non-coding rnas |
| EP2718439B1 (en) * | 2011-06-09 | 2017-08-09 | CuRNA, Inc. | Treatment of frataxin (fxn) related diseases by inhibition of natural antisense transcript to fxn |
| JP2015523854A (ja) | 2012-05-16 | 2015-08-20 | ラナ セラピューティクス インコーポレイテッド | Smn遺伝子ファミリー発現を調節するための組成物及び方法 |
| WO2013173652A1 (en) | 2012-05-16 | 2013-11-21 | Rana Therapeutics, Inc. | Compositions and methods for modulating gene expression |
| AU2013262709A1 (en) | 2012-05-16 | 2015-01-22 | Rana Therapeutics, Inc. | Compositions and methods for modulating MECP2 expression |
| US10837014B2 (en) | 2012-05-16 | 2020-11-17 | Translate Bio Ma, Inc. | Compositions and methods for modulating SMN gene family expression |
| CA2873769A1 (en) | 2012-05-16 | 2013-11-21 | Rana Therapeutics Inc. | Compositions and methods for modulating hemoglobin gene family expression |
| CA2873766A1 (en) | 2012-05-16 | 2013-11-21 | Rana Therapeutics Inc. | Compositions and methods for modulating atp2a2 expression |
| BR112014028634A2 (pt) | 2012-05-16 | 2017-06-27 | Rana Therapeutics Inc | composições e métodos para modulação da expressão de utrn |
| ES3047691T3 (en) | 2012-08-01 | 2025-12-04 | Ikaika Therapeutics Inc | Mitigating tissue damage and fibrosis via latent transforming growth factor beta binding protein (ltbp4) |
| CN103405774A (zh) * | 2012-10-24 | 2013-11-27 | 尹海芳 | 一种新型药物靶向运输辅助剂 |
| KR101485576B1 (ko) * | 2013-05-29 | 2015-01-21 | 고려대학교 산학협력단 | 신트로핀 베타2 siRNA를 발현하는 살모넬라 균주 및 이를 함유하는 항암제 조성물 |
| KR101666618B1 (ko) * | 2013-08-16 | 2016-10-14 | 아주대학교산학협력단 | 디스트로핀 폴리뉴클레오티드의 발현을 억제하는 뉴클레오티드가 도입된 동물 세포주 |
| GB201410693D0 (en) | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
| CN107109411B (zh) | 2014-10-03 | 2022-07-01 | 冷泉港实验室 | 核基因输出的定向增加 |
| CA2966044A1 (en) | 2014-10-30 | 2016-05-06 | The General Hospital Corporation | Methods for modulating atrx-dependent gene repression |
| WO2016149455A2 (en) | 2015-03-17 | 2016-09-22 | The General Hospital Corporation | The rna interactome of polycomb repressive complex 1 (prc1) |
| JP7511326B2 (ja) | 2015-10-09 | 2024-07-05 | ウェイブ ライフ サイエンシズ リミテッド | オリゴヌクレオチド組成物およびその方法 |
| EP3359685B1 (en) | 2015-10-09 | 2026-01-28 | University Of Southampton | Modulation of gene expression for deregulated protein expression |
| EP3933041B1 (en) | 2015-12-14 | 2024-01-31 | Cold Spring Harbor Laboratory | Antisense oligomers for treatment of autosomal dominant retardation |
| CN106248953A (zh) * | 2016-07-14 | 2016-12-21 | 武汉璟泓万方堂医药科技股份有限公司 | 一种免疫层析试纸卡及其制备方法和用途 |
| SG11202001590RA (en) | 2017-08-25 | 2020-03-30 | Stoke Therapeutics Inc | Antisense oligomers for treatment of conditions and diseases |
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| EP3829595A4 (en) | 2018-08-02 | 2022-08-24 | Dyne Therapeutics, Inc. | MUSCLE-DIRECTED COMPLEXES AND USES THEREOF IN THE TREATMENT OF DYSTROPHINOPATHIES |
| US20220193250A1 (en) | 2018-08-02 | 2022-06-23 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US12018087B2 (en) | 2018-08-02 | 2024-06-25 | Dyne Therapeutics, Inc. | Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject |
| CA3124415A1 (en) | 2018-12-21 | 2020-06-25 | Northwestern University | Use of annexins in preventing and treating muscle membrane injury |
| US20220062299A1 (en) | 2018-12-26 | 2022-03-03 | Northwestern University | Use of glucocorticoid steroids in preventing and treating conditions of muscle wasting, aging and metabolic disorder |
| KR20210134003A (ko) | 2019-02-27 | 2021-11-08 | 스톡 테라퓨틱스, 인크. | 병태 및 질환의 치료를 위한 안티센스 올리고머 |
| CA3138945A1 (en) * | 2019-05-06 | 2020-11-12 | Antisense Therapeutics Ltd | Methods for treating muscular dystrophy using inhibitory oligonucleotides to cd49d |
| JP7780691B2 (ja) * | 2020-01-10 | 2025-12-05 | ダイン セラピューティクス,インコーポレーテッド | ジストロフィン異常症を処置するための筋標的化複合体およびそれらの使用 |
| BR112022022889A2 (pt) | 2020-05-11 | 2023-04-04 | Stoke Therapeutics Inc | Oligômeros antissentido de opa1 para tratamento de condições e doenças |
| IL309936A (en) | 2021-07-09 | 2024-03-01 | Dyne Therapeutics Inc | Muscle targeting complexes and formulations for the treatment of dystrophinopathy |
| US11969475B2 (en) | 2021-07-09 | 2024-04-30 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
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| CN118829728A (zh) * | 2022-01-20 | 2024-10-22 | 中美瑞康核酸技术(南通)研究院有限公司 | 活化抗肌萎缩蛋白相关蛋白表达的寡核苷酸调节剂 |
| CN115820642B (zh) * | 2022-11-11 | 2023-10-10 | 昆明理工大学 | 一种用于治疗杜氏肌营养不良症的CRISPR-Cas9系统 |
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| US20120046345A1 (en) | 2012-02-23 |
| CN102459597A (zh) | 2012-05-16 |
| US9533004B2 (en) | 2017-01-03 |
| CA3185821A1 (en) | 2010-11-11 |
| KR101742334B1 (ko) | 2017-06-01 |
| WO2010129861A2 (en) | 2010-11-11 |
| US20150094356A1 (en) | 2015-04-02 |
| US9012139B2 (en) | 2015-04-21 |
| WO2010129861A3 (en) | 2011-05-12 |
| CA2761248A1 (en) | 2010-11-11 |
| EP2427554B1 (en) | 2016-11-16 |
| EP2427554A4 (en) | 2013-05-22 |
| ES2618572T3 (es) | 2017-06-21 |
| EP2427554A2 (en) | 2012-03-14 |
| CA2761248C (en) | 2023-03-14 |
| KR20120013425A (ko) | 2012-02-14 |
| CN102459597B (zh) | 2020-06-30 |
| JP2012525852A (ja) | 2012-10-25 |
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