CN105838714B - 反义分子和治疗疾病的方法 - Google Patents
反义分子和治疗疾病的方法 Download PDFInfo
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- CN105838714B CN105838714B CN201610091098.5A CN201610091098A CN105838714B CN 105838714 B CN105838714 B CN 105838714B CN 201610091098 A CN201610091098 A CN 201610091098A CN 105838714 B CN105838714 B CN 105838714B
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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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- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
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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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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/11—Antisense
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- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
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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
- C12N2310/31—Chemical structure of the backbone
- C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
- C12N2310/3181—Peptide nucleic acid, PNA
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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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
- C12N2310/3233—Morpholino-type ring
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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
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- 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
- C12N2310/3521—Methyl
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/33—Alteration of splicing
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- Engineering & Computer Science (AREA)
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- Biomedical Technology (AREA)
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- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
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- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- Epidemiology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| AU2009905549A AU2009905549A0 (en) | 2009-11-12 | Antisense Molecules and Methods for Treating Pathologies | |
| AU2009905549 | 2009-11-12 | ||
| CN2010800613405A CN103003430A (zh) | 2009-11-12 | 2010-11-12 | 反义分子和治疗疾病的方法 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN2010800613405A Division CN103003430A (zh) | 2009-11-12 | 2010-11-12 | 反义分子和治疗疾病的方法 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| CN105838714A CN105838714A (zh) | 2016-08-10 |
| CN105838714B true CN105838714B (zh) | 2020-07-17 |
Family
ID=43991096
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN201610091098.5A Active CN105838714B (zh) | 2009-11-12 | 2010-11-12 | 反义分子和治疗疾病的方法 |
| CN2010800613405A Pending CN103003430A (zh) | 2009-11-12 | 2010-11-12 | 反义分子和治疗疾病的方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN2010800613405A Pending CN103003430A (zh) | 2009-11-12 | 2010-11-12 | 反义分子和治疗疾病的方法 |
Country Status (23)
| Country | Link |
|---|---|
| US (7) | US8637483B2 (enExample) |
| EP (2) | EP3431603A1 (enExample) |
| JP (6) | JP5963678B2 (enExample) |
| KR (9) | KR102000762B1 (enExample) |
| CN (2) | CN105838714B (enExample) |
| AU (5) | AU2010317599B2 (enExample) |
| BR (2) | BR122023023194A2 (enExample) |
| CA (1) | CA2780563C (enExample) |
| CY (1) | CY1121198T1 (enExample) |
| DK (1) | DK2499249T3 (enExample) |
| ES (1) | ES2693459T3 (enExample) |
| HR (1) | HRP20181824T1 (enExample) |
| HU (1) | HUE040445T2 (enExample) |
| IL (6) | IL297299A (enExample) |
| LT (1) | LT2499249T (enExample) |
| NZ (2) | NZ716534A (enExample) |
| PL (1) | PL2499249T3 (enExample) |
| PT (1) | PT2499249T (enExample) |
| RS (1) | RS58079B1 (enExample) |
| SI (1) | SI2499249T1 (enExample) |
| SM (1) | SMT201800579T1 (enExample) |
| TR (1) | TR201816523T4 (enExample) |
| WO (1) | WO2011057350A1 (enExample) |
Families Citing this family (104)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2003225410A1 (en) | 2003-03-21 | 2004-10-11 | Academisch Ziekenhuis Leiden | Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure |
| USRE48960E1 (en) | 2004-06-28 | 2022-03-08 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| EP1766010B1 (en) | 2004-06-28 | 2011-02-16 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| ES2852549T3 (es) | 2005-02-09 | 2021-09-13 | Sarepta Therapeutics Inc | Composición antisentido para tratamiento de la atrofia muscular |
| PL2203173T3 (pl) | 2007-10-26 | 2016-06-30 | Academisch Ziekenhuis Leiden | Środki i sposoby przeciwdziałania zaburzeniom mięśni |
| USRE48468E1 (en) | 2007-10-26 | 2021-03-16 | Biomarin Technologies B.V. | Means and methods for counteracting muscle disorders |
| EP2119783A1 (en) | 2008-05-14 | 2009-11-18 | Prosensa Technologies B.V. | Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means |
| KR20180118828A (ko) | 2008-10-24 | 2018-10-31 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | Dmd를 위한 다중 엑손 스키핑 조성물 |
| EP3431603A1 (en) | 2009-11-12 | 2019-01-23 | The University Of Western Australia | Antisense molecules and methods for treating pathologies |
| JP6141018B2 (ja) | 2009-12-24 | 2017-06-07 | バイオマリン テクノロジーズ ベー.フェー. | 炎症性障害を治療するための分子 |
| TWI541024B (zh) | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
| EP2672977A1 (en) | 2011-02-08 | 2013-12-18 | The Charlotte-Mecklenburg Hospital Authority d/b/a Carolina Healthcare System | Antisense oligonucleotides |
| US20130085139A1 (en) | 2011-10-04 | 2013-04-04 | Royal Holloway And Bedford New College | Oligomers |
| CN104024414A (zh) | 2011-12-28 | 2014-09-03 | 日本新药株式会社 | 反义核酸 |
| JP6928025B2 (ja) * | 2012-01-27 | 2021-09-01 | バイオマリン テクノロジーズ ベー.フェー. | デュシェンヌ型及びベッカー型筋ジストロフィーの治療のための改善された特徴を有するrna調節オリゴヌクレオチド |
| JP2015509922A (ja) * | 2012-01-27 | 2015-04-02 | プロセンサ テクノロジーズ ビー.ブイ.Prosensa Technologies B.V. | デュシェンヌ型及びベッカー型筋ジストロフィーの治療のための改善された特徴を有するrna調節オリゴヌクレオチド |
| DE102012103041A1 (de) * | 2012-04-10 | 2013-10-10 | Eberhard-Karls-Universität Tübingen Universitätsklinikum | Behandlung der dilativen Kardiomyopathie mittels Antisense-Oligonucleotiden |
| CN110257379B (zh) * | 2012-07-03 | 2023-08-11 | 马林生物科技有限公司 | 用于治疗肌肉萎缩症患者的寡核苷酸 |
| EA201591178A1 (ru) * | 2012-12-20 | 2015-11-30 | Сарепта Терапьютикс, Инк. | Улучшенные композиции для пропускания экзона для лечения мышечной дистрофии |
| CN110066793A (zh) * | 2013-03-14 | 2019-07-30 | 萨勒普塔医疗公司 | 用于治疗肌肉萎缩的外显子跳跃组合物 |
| KR20230116945A (ko) * | 2013-03-14 | 2023-08-04 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이영양증의 치료를 위한 엑손 스키핑 조성물 |
| BR112015022998A2 (pt) | 2013-03-15 | 2017-11-14 | Sarepta Therapeutics Inc | composições melhoradas para o tratamento de distrofia muscular |
| CA2909807C (en) | 2013-04-20 | 2023-08-08 | Research Institute At Nationwide Children's Hospital | Recombinant adeno-associated virus delivery of exon 2-targeted u7snrna polynucleotide constructs |
| MX2016002044A (es) * | 2013-08-16 | 2016-08-17 | Rana Therapeutics Inc | Composiciones y metodos para modular el acido ribonucleico. |
| LT3118311T (lt) * | 2014-03-12 | 2019-04-10 | Nippon Shinyaku Co., Ltd. | Priešprasminė nukleorūgštis |
| GB201410693D0 (en) | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
| MY194170A (en) | 2014-06-17 | 2022-11-16 | Nippon Shinyaku Co Ltd | Antisense nucleic acids |
| JP6986444B2 (ja) * | 2014-08-09 | 2021-12-22 | リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル | Dmd遺伝子のエクソン5内の内部リボソーム進入部位を活性化するための方法及び材料 |
| WO2016025469A1 (en) * | 2014-08-11 | 2016-02-18 | The Board Of Regents Of The University Of Texas System | Prevention of muscular dystrophy by crispr/cas9-mediated gene editing |
| KR20170045344A (ko) | 2014-09-07 | 2017-04-26 | 셀렉타 바이오사이언시즈, 인크. | 항-바이러스 전달 벡터 면역 반응을 약화시키기 위한 방법 및 조성물 |
| CA2963288A1 (en) | 2014-10-03 | 2016-04-07 | Cold Spring Harbor Laboratory | Targeted augmentation of nuclear gene output |
| EP3256591A4 (en) | 2015-02-13 | 2018-08-08 | Translate Bio Ma, Inc. | Hybrid oligonucleotides and uses thereof |
| MA41795A (fr) | 2015-03-18 | 2018-01-23 | Sarepta Therapeutics Inc | Exclusion d'un exon induite par des composés antisens dans la myostatine |
| LT3351633T (lt) | 2015-09-15 | 2020-08-10 | Nippon Shinyaku Co., Ltd. | Priešprasmė nukleorūgštis |
| WO2017062835A2 (en) | 2015-10-09 | 2017-04-13 | Sarepta Therapeutics, Inc. | Compositions and methods for treating duchenne muscular dystrophy and related disorders |
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| AU2016334804B2 (en) | 2015-10-09 | 2022-03-31 | University Of Southampton | Modulation of gene expression and screening for deregulated protein expression |
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| FR3044926B1 (fr) * | 2015-12-09 | 2020-01-31 | Genethon | Outils de therapie genique efficaces pour le saut de l'exon 53 de la dystrophine |
| WO2017106377A1 (en) | 2015-12-14 | 2017-06-22 | Cold Spring Harbor Laboratory | Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| JOP20200228A1 (ar) | 2015-12-21 | 2017-06-16 | Novartis Ag | تركيبات وطرق لخفض تعبير البروتين tau |
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