JP2015523854A - 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 - Google Patents
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 Download PDFInfo
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- JP2015523854A JP2015523854A JP2015512858A JP2015512858A JP2015523854A JP 2015523854 A JP2015523854 A JP 2015523854A JP 2015512858 A JP2015512858 A JP 2015512858A JP 2015512858 A JP2015512858 A JP 2015512858A JP 2015523854 A JP2015523854 A JP 2015523854A
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- stranded oligonucleotide
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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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- 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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- 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
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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
- A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
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- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
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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
- C12N15/1131—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 against viruses
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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
- C12N15/1131—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 against viruses
- C12N15/1132—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 against viruses against retroviridae, e.g. HIV
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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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
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
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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
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3517—Marker; Tag
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- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Epidemiology (AREA)
- Virology (AREA)
- AIDS & HIV (AREA)
- Pain & Pain Management (AREA)
- Neurology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261647858P | 2012-05-16 | 2012-05-16 | |
| US61/647,858 | 2012-05-16 | ||
| US201261719394P | 2012-10-27 | 2012-10-27 | |
| US61/719,394 | 2012-10-27 | ||
| US201361785529P | 2013-03-14 | 2013-03-14 | |
| US61/785,529 | 2013-03-14 | ||
| PCT/US2013/041440 WO2013173638A1 (en) | 2012-05-16 | 2013-05-16 | Compositions and methods for modulating smn gene family expression |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2015523854A true JP2015523854A (ja) | 2015-08-20 |
| JP2015523854A5 JP2015523854A5 (enExample) | 2016-06-30 |
Family
ID=49584305
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015512858A Pending JP2015523854A (ja) | 2012-05-16 | 2013-05-16 | 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 |
Country Status (13)
| Country | Link |
|---|---|
| US (1) | US10059941B2 (enExample) |
| EP (1) | EP2850186B1 (enExample) |
| JP (1) | JP2015523854A (enExample) |
| KR (1) | KR20150030205A (enExample) |
| CN (1) | CN104540947A (enExample) |
| AU (1) | AU2013262649A1 (enExample) |
| CA (1) | CA2873794A1 (enExample) |
| DK (1) | DK2850186T3 (enExample) |
| EA (1) | EA201492123A1 (enExample) |
| HK (1) | HK1208700A1 (enExample) |
| SG (1) | SG11201407483YA (enExample) |
| WO (1) | WO2013173638A1 (enExample) |
| ZA (1) | ZA201409228B (enExample) |
Cited By (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2018525015A (ja) * | 2015-08-28 | 2018-09-06 | サレプタ セラピューティクス,インコーポレイテッド | 脊髄性筋萎縮症におけるエクソン包含のための修飾アンチセンスオリゴマー |
| US11584932B2 (en) | 2016-11-01 | 2023-02-21 | The Research Foundation For The State University Of New York | 5-halouracil-modified microRNAs and their use in the treatment of cancer |
| US12319913B2 (en) | 2018-10-31 | 2025-06-03 | The Research Foundation For The State University Of New York | 5-halouracil-modified microRNAs and their use in the treatment of cancer |
| US12458604B2 (en) | 2020-10-14 | 2025-11-04 | The Trustees Of The University Of Pennsylvania | Methods of lipid nanoparticle manufacture and compositions derived therefrom |
Families Citing this family (38)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9920317B2 (en) | 2010-11-12 | 2018-03-20 | The General Hospital Corporation | Polycomb-associated non-coding RNAs |
| EP2638163B1 (en) | 2010-11-12 | 2017-05-17 | The General Hospital Corporation | Polycomb-associated non-coding rnas |
| EA201492120A1 (ru) | 2012-05-16 | 2015-10-30 | Рана Терапьютикс, Инк. | Композиции и способы для модулирования экспрессии atp2a2 |
| WO2013173638A1 (en) | 2012-05-16 | 2013-11-21 | Rana Therapeutics, Inc. | Compositions and methods for modulating smn gene family 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 |
| KR20160074368A (ko) | 2012-05-16 | 2016-06-28 | 라나 테라퓨틱스, 인크. | Utrn 발현을 조절하기 위한 조성물 및 방법 |
| BR112014028631A2 (pt) | 2012-05-16 | 2017-10-17 | Rana Therapeutics Inc | composições e métodos para modulação da expressão da família de genes da hemoglobina |
| US9885040B2 (en) | 2013-04-12 | 2018-02-06 | The Curators Of The University Of Missouri | SMN2 element 1 antisense compositions and methods and uses thereof |
| WO2015051283A1 (en) | 2013-10-04 | 2015-04-09 | Rana Therapeutics, Inc. | Compositions and methods for treating amyotrophic lateral sclerosis |
| CN103911346B (zh) * | 2014-03-27 | 2016-09-07 | 江苏雄鸣医药科技有限公司 | 一种用于治疗脊髓性肌萎缩症药物筛选的细胞模型 |
| US10436802B2 (en) | 2014-09-12 | 2019-10-08 | Biogen Ma Inc. | Methods for treating spinal muscular atrophy |
| WO2016070060A1 (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) |
| WO2016164896A2 (en) | 2015-04-10 | 2016-10-13 | Ionis Pharmaceuticals, Inc. | Modulation of smn expression |
| US11249071B2 (en) | 2015-04-24 | 2022-02-15 | California Institute Of Technology | Reactivation of x chromosome genes |
| US20180312839A1 (en) * | 2015-10-26 | 2018-11-01 | Translate Bio Ma, Inc. | Methods and compositions for increasing smn expression |
| WO2017087486A1 (en) | 2015-11-16 | 2017-05-26 | Ohio State Innovation Foundation | Methods and compositions for treating disorders and diseases using survival motor neuron (smn) protein |
| EP3471779A4 (en) * | 2016-06-16 | 2020-07-08 | Ionis Pharmaceuticals, Inc. | COMBINATIONS FOR MODULATING SMN EXPRESSION |
| JP2019537427A (ja) | 2016-10-27 | 2019-12-26 | カリフォルニア インスティチュート オブ テクノロジー | X染色体の再活性化のためのhdac阻害剤組成物 |
| AU2017368050A1 (en) | 2016-11-29 | 2019-06-20 | Puretech Lyt, Inc. | Exosomes for delivery of therapeutic agents |
| EP3599844A4 (en) * | 2017-06-07 | 2021-01-13 | University of Massachusetts | ANTI-ADAM33 OLIGONUCLEOTIDES AND RELATED PROCESSES |
| WO2019048631A1 (en) | 2017-09-08 | 2019-03-14 | Mina Therapeutics Limited | SMALL HNF4A ACTIVATOR RNA COMPOSITIONS AND METHODS OF USE |
| US20200208152A1 (en) | 2017-09-08 | 2020-07-02 | Mina Therapeutics Limited | Stabilized sarna compositions and methods of use |
| WO2019075357A1 (en) * | 2017-10-12 | 2019-04-18 | Wave Life Sciences Ltd. | OLIGONUCLEOTIDE COMPOSITIONS AND RELATED METHODS |
| US11185079B2 (en) | 2018-09-26 | 2021-11-30 | Greenlight Biosciences, Inc. | Control of Coleopteran insects |
| AU2019377855A1 (en) | 2018-11-08 | 2021-06-03 | Greenlight Biosciences, Inc. | Control of insect infestation |
| WO2020146519A1 (en) * | 2019-01-09 | 2020-07-16 | Coyote Bioscience Usa Inc. | Methods and systems for identification of spinal muscular atrophy |
| WO2021030766A1 (en) * | 2019-08-15 | 2021-02-18 | Biogen Ma Inc. | Combination therapy for spinal muscular atrophy |
| JP2022545101A (ja) | 2019-08-19 | 2022-10-25 | ミナ セラピューティクス リミテッド | オリゴヌクレオチドコンジュゲート組成物および使用方法 |
| JP7446443B2 (ja) | 2020-02-28 | 2024-03-08 | アイオーニス ファーマシューティカルズ, インコーポレーテッド | Smn2を調節するための化合物及び方法 |
| CN113444722B (zh) * | 2020-03-24 | 2024-09-24 | 中国科学院脑科学与智能技术卓越创新中心 | 单碱基编辑介导的剪接修复在制备治疗脊髓性肌萎缩症中的应用 |
| AU2021307774A1 (en) * | 2020-07-13 | 2023-03-02 | Cold Spring Harbor Laboratory | Compositions for treatment of spinal muscular atrophy |
| EP4294459A4 (en) * | 2021-02-19 | 2025-06-18 | University Of Florida Research Foundation, Incorporated | Methods and compositions to confer regulation to gene therapy cargoes by heterologous use of alternative splicing cassettes |
| WO2023020421A1 (en) * | 2021-08-16 | 2023-02-23 | Hangzhou Jiayin Biotech Ltd. | Regulation of imprinting silenced genes by smn1 and snrpn expression and uses thereof |
| EP4396353A2 (en) * | 2021-09-03 | 2024-07-10 | Camp4 Therapeutics Corporation | Modulation of gene transcription using antisense oligonucleotides targeting regulatory rnas |
| AU2022421244A1 (en) | 2021-12-22 | 2024-07-11 | Camp4 Therapeutics Corporation | Modulation of gene transcription using antisense oligonucleotides targeting regulatory rnas |
| AU2024208843A1 (en) * | 2023-01-19 | 2025-08-14 | Research Institute At Nationwide Children's Hospital | Materials and methods for modulating expression |
Citations (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20070292408A1 (en) * | 2004-12-03 | 2007-12-20 | University Of Massachusetts | Spinal Muscular Atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences |
| WO2008103763A2 (en) * | 2007-02-20 | 2008-08-28 | Sequenom, Inc. | Methods and compositions for cancer diagnosis and treatment based on nucleic acid methylation |
| WO2012027033A1 (en) * | 2010-07-19 | 2012-03-01 | Isis Pharmaceuticals, Inc. | Compounds and methods for modulating target nuclear and sub-nuclear nucleic acid molecules in cells and animals |
| WO2012065143A1 (en) * | 2010-11-12 | 2012-05-18 | The General Hospital Corporation | Polycomb-associated non-coding rnas |
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| JP2018525015A (ja) * | 2015-08-28 | 2018-09-06 | サレプタ セラピューティクス,インコーポレイテッド | 脊髄性筋萎縮症におけるエクソン包含のための修飾アンチセンスオリゴマー |
| US11584932B2 (en) | 2016-11-01 | 2023-02-21 | The Research Foundation For The State University Of New York | 5-halouracil-modified microRNAs and their use in the treatment of cancer |
| US12371693B2 (en) | 2016-11-01 | 2025-07-29 | The Research Foundation For The State University Of New York | 5-halouracil-modified micrornas and their use in the treatment of cancer |
| US12319913B2 (en) | 2018-10-31 | 2025-06-03 | The Research Foundation For The State University Of New York | 5-halouracil-modified microRNAs and their use in the treatment of cancer |
| US12458604B2 (en) | 2020-10-14 | 2025-11-04 | The Trustees Of The University Of Pennsylvania | Methods of lipid nanoparticle manufacture and compositions derived therefrom |
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| EP2850186A4 (en) | 2016-01-27 |
| ZA201409228B (en) | 2016-07-27 |
| CA2873794A1 (en) | 2013-11-21 |
| WO2013173638A1 (en) | 2013-11-21 |
| KR20150030205A (ko) | 2015-03-19 |
| US20150252364A1 (en) | 2015-09-10 |
| EP2850186B1 (en) | 2018-12-19 |
| US10059941B2 (en) | 2018-08-28 |
| CN104540947A (zh) | 2015-04-22 |
| SG11201407483YA (en) | 2014-12-30 |
| EA201492123A1 (ru) | 2015-10-30 |
| DK2850186T3 (en) | 2019-04-08 |
| AU2013262649A1 (en) | 2015-01-22 |
| EP2850186A1 (en) | 2015-03-25 |
| HK1208700A1 (en) | 2016-03-11 |
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