CN116585344A - 用于治疗奥尔波特综合征的方法 - Google Patents
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Classifications
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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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- A61K9/00—Medicinal preparations characterised by special physical form
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- 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
- A61K9/0021—Intradermal administration, e.g. through microneedle arrays, needleless injectors
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- A—HUMAN NECESSITIES
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- A61P13/00—Drugs for disorders of the urinary system
- A61P13/12—Drugs for disorders of the urinary system of the kidneys
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- 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
-
- 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
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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
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
- C12N2310/141—MicroRNAs, miRNAs
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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
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
-
- 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
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- 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
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3525—MOE, methoxyethoxy
Landscapes
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- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
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- Epidemiology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Biochemistry (AREA)
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- Dermatology (AREA)
- Physics & Mathematics (AREA)
- General Chemical & Material Sciences (AREA)
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- Plant Pathology (AREA)
- Microbiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Urology & Nephrology (AREA)
- Inorganic Chemistry (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Investigating Or Analysing Biological Materials (AREA)
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| US201762501699P | 2017-05-04 | 2017-05-04 | |
| US62/501,699 | 2017-05-04 | ||
| CN201880037914.1A CN110740737A (zh) | 2017-05-04 | 2018-05-04 | 用于治疗奥尔波特综合征的方法 |
| PCT/US2018/031094 WO2018204788A1 (en) | 2017-05-04 | 2018-05-04 | Methods for treatment of alport syndrome |
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| WO2022076452A1 (en) * | 2020-10-05 | 2022-04-14 | Father Flanagan's Boys' Home Doing Business As Boys Town National Research Hospital | Combination therapy for alport renal disease |
| CN120731923B (zh) * | 2025-09-05 | 2025-11-04 | 内蒙古医科大学附属医院(内蒙古自治区心血管研究所) | 一种Alport综合征小鼠NMD逃逸模型的构建方法及其应用 |
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| US20080152700A1 (en) * | 2004-06-01 | 2008-06-26 | Reza Sheikhnejad | Methods and compositions for the inhibition of gene expression |
| KR101877698B1 (ko) * | 2008-08-25 | 2018-07-12 | 엑스칼리아드 파마슈티컬즈, 인코포레이티드 | 결합 조직 성장 인자에 대한 안티센스 올리고뉴클레오타이드 및 그의 용도 |
| EP2825648B1 (en) * | 2012-03-15 | 2018-09-05 | CuRNA, Inc. | Treatment of brain derived neurotrophic factor (bdnf) related diseases by inhibition of natural antisense transcript to bdnf |
| BR112014026639A8 (pt) * | 2012-04-25 | 2018-01-16 | Regulus Therapeutics Inc | compostos , métodos de inibição da atividade de mir-21 , método para diminuir a expressão de colágeno , método para tratar , prevenir ou atrasar o início de uma doença , método de tratamento de um distúrbio fibroproliferativo e usos de um composto. |
| UA116639C2 (uk) * | 2012-10-09 | 2018-04-25 | Рег'Юлес Терап'Ютікс Інк. | Способи лікування синдрому альпорта |
| GB201507926D0 (en) * | 2015-05-08 | 2015-06-24 | Proqr Therapeutics N V | Improved treatments using oligonucleotides |
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| CN110740737A (zh) | 2020-01-31 |
| WO2018204788A1 (en) | 2018-11-08 |
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| KR20190141770A (ko) | 2019-12-24 |
| CL2019003153A1 (es) | 2020-03-27 |
| CA3062316A1 (en) | 2018-11-08 |
| AU2018261792A1 (en) | 2020-01-02 |
| RU2019139261A (ru) | 2021-06-04 |
| BR112019022824A2 (pt) | 2020-06-09 |
| US20210100828A1 (en) | 2021-04-08 |
| MX2019013158A (es) | 2020-02-07 |
| MA50749A (fr) | 2020-03-11 |
| JP2023075111A (ja) | 2023-05-30 |
| US20220133769A1 (en) | 2022-05-05 |
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