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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- 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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- 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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- 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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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
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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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- 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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- 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
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
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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
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3517—Marker; Tag
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- Genetics & Genomics (AREA)
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- Organic Chemistry (AREA)
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- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
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- Zoology (AREA)
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- General Engineering & Computer Science (AREA)
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- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
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- Virology (AREA)
- Neurology (AREA)
- Pain & Pain Management (AREA)
- AIDS & HIV (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)
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- 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 (40)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3702460A1 (en) | 2010-11-12 | 2020-09-02 | The General Hospital Corporation | Polycomb-associated non-coding rnas |
| US9920317B2 (en) | 2010-11-12 | 2018-03-20 | The General Hospital Corporation | Polycomb-associated non-coding RNAs |
| CN104583402A (zh) | 2012-05-16 | 2015-04-29 | Rana医疗有限公司 | 用于调节mecp2表达的组合物和方法 |
| 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 |
| EA201492120A1 (ru) | 2012-05-16 | 2015-10-30 | Рана Терапьютикс, Инк. | Композиции и способы для модулирования экспрессии atp2a2 |
| US10174315B2 (en) | 2012-05-16 | 2019-01-08 | The General Hospital Corporation | Compositions and methods for modulating hemoglobin gene family expression |
| US10837014B2 (en) | 2012-05-16 | 2020-11-17 | Translate Bio Ma, Inc. | Compositions and methods for modulating SMN gene family expression |
| CN104540946A (zh) | 2012-05-16 | 2015-04-22 | Rana医疗有限公司 | 用于调节utrn表达的组合物和方法 |
| 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 |
| EP3052632A4 (en) | 2013-10-04 | 2017-03-29 | 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 |
| WO2016040748A1 (en) | 2014-09-12 | 2016-03-17 | Ionis Pharmaceuticals, Inc. | Compositions and methods for detection of smn protein in a subject and treatment of a subject |
| WO2016070060A1 (en) | 2014-10-30 | 2016-05-06 | The General Hospital Corporation | Methods for modulating atrx-dependent gene repression |
| US10900036B2 (en) | 2015-03-17 | 2021-01-26 | The General Hospital Corporation | RNA interactome of polycomb repressive complex 1 (PRC1) |
| US10851371B2 (en) | 2015-04-10 | 2020-12-01 | Ionis Pharmaceuticals, Inc. | Modulation of SMN expression |
| WO2016172734A1 (en) | 2015-04-24 | 2016-10-27 | California Institute Of Technology | Reactivation of x chromosome genes |
| WO2017075030A1 (en) * | 2015-10-26 | 2017-05-04 | Rana Therapeutics, Inc. | Methods and compositions for increasing smn expression |
| US10357543B2 (en) | 2015-11-16 | 2019-07-23 | Ohio State Innovation Foundation | Methods and compositions for treating disorders and diseases using Survival Motor Neuron (SMN) protein |
| US11198867B2 (en) | 2016-06-16 | 2021-12-14 | Ionis Pharmaceuticals, Inc. | Combinations for the modulation of 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 |
| US20180193270A1 (en) | 2016-11-29 | 2018-07-12 | PureTech Health LLC | Exosomes for delivery of therapeutic agents |
| US11401519B2 (en) | 2017-06-07 | 2022-08-02 | University Of Massachusetts | Anti-ADAM33 oligonucleotides and related methods |
| WO2019048632A1 (en) | 2017-09-08 | 2019-03-14 | Mina Therapeutics Limited | STABILIZED COMPOSITIONS OF SMALL ACTIVATORY RNA (PARNA) OF HNF4A AND METHODS OF USE |
| WO2019048645A1 (en) | 2017-09-08 | 2019-03-14 | Mina Therapeutics Limited | STABILIZED COMPOSITIONS OF SMALL ACTIVATOR RNA (PARNA) FROM CEBPA AND METHODS OF USE |
| EP3694530A4 (en) * | 2017-10-12 | 2021-06-30 | Wave Life Sciences Ltd. | COMPOSITIONS OF OLIGONUCLEOTIDES AND RELATED PROCESSES |
| JP2022502049A (ja) | 2018-09-26 | 2022-01-11 | グリーンライト バイオサイエンシーズ インコーポレーテッドGreenlight Biosciences, Inc. | Coleopteran昆虫の制御 |
| WO2020097414A1 (en) | 2018-11-08 | 2020-05-14 | Greenlight Biosciences, Inc. | Control of insect infestation |
| BR112021013612A2 (pt) * | 2019-01-09 | 2021-09-28 | Coyote Bioscience Usa Inc. | Métodos e sistemas para a identificação de atrofia muscular espinhal |
| CN111849968A (zh) | 2019-04-30 | 2020-10-30 | 中美瑞康核酸技术(南通)研究院有限公司 | 寡核苷酸分子及其在急性间歇性卟啉症治疗中的应用 |
| EP4013387A4 (en) * | 2019-08-15 | 2023-09-27 | Biogen MA Inc. | COMBINATION THERAPY FOR SPINAL MUSCLE ATROPHY |
| CN114585633A (zh) | 2019-08-19 | 2022-06-03 | 米纳治疗有限公司 | 寡核苷酸缀合物组合物和使用方法 |
| AU2021225957A1 (en) | 2020-02-28 | 2022-09-08 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating SMN2 |
| 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 |
| US20230302036A1 (en) * | 2020-07-13 | 2023-09-28 | Consejo Nacional De Investigaciones Cientificas Y Tecnicas | Compositions for treatment of spinal muscular atrophy |
| GB202101187D0 (en) * | 2021-01-28 | 2021-03-17 | Univ Pretoria | An immunogen |
| US20240141384A1 (en) * | 2021-02-19 | 2024-05-02 | University Of Florida Research Foundation, Incoporated | 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 |
| EP4396353A4 (en) * | 2021-09-03 | 2026-02-25 | Camp4 Therapeutics Corp | MODULATION OF GENE TRANSCRIPTION USING ANTISENSE OLIGONUCLEOTIDES TARGETING REGULATOR RNAS |
| WO2023122762A1 (en) | 2021-12-22 | 2023-06-29 | Camp4 Therapeutics Corporation | Modulation of gene transcription using antisense oligonucleotides targeting regulatory rnas |
| WO2024155792A2 (en) * | 2023-01-19 | 2024-07-25 | 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 |
| US12576040B2 (en) | 2020-10-14 | 2026-03-17 | The Trustees Of The University Of Pennsylvania | Ionizable lipids and methods of manufacture and use thereof |
Also Published As
| Publication number | Publication date |
|---|---|
| WO2013173638A1 (en) | 2013-11-21 |
| HK1208700A1 (en) | 2016-03-11 |
| US20150252364A1 (en) | 2015-09-10 |
| US10059941B2 (en) | 2018-08-28 |
| EA201492123A1 (ru) | 2015-10-30 |
| SG11201407483YA (en) | 2014-12-30 |
| KR20150030205A (ko) | 2015-03-19 |
| AU2013262649A1 (en) | 2015-01-22 |
| ZA201409228B (en) | 2016-07-27 |
| CN104540947A (zh) | 2015-04-22 |
| EP2850186A4 (en) | 2016-01-27 |
| DK2850186T3 (en) | 2019-04-08 |
| CA2873794A1 (en) | 2013-11-21 |
| EP2850186B1 (en) | 2018-12-19 |
| EP2850186A1 (en) | 2015-03-25 |
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