JP4974890B2 - ペプチドが結合された、イノシン置換アンチセンスオリゴマー化合物および方法 - Google Patents
ペプチドが結合された、イノシン置換アンチセンスオリゴマー化合物および方法 Download PDFInfo
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- JP4974890B2 JP4974890B2 JP2007527563A JP2007527563A JP4974890B2 JP 4974890 B2 JP4974890 B2 JP 4974890B2 JP 2007527563 A JP2007527563 A JP 2007527563A JP 2007527563 A JP2007527563 A JP 2007527563A JP 4974890 B2 JP4974890 B2 JP 4974890B2
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- 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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- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
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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
- C12N15/1135—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 oncogenes or tumor suppressor genes
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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
- C12N2310/314—Phosphoramidates
- C12N2310/3145—Phosphoramidates with the nitrogen in 3' or 5'-position
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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
- C12N2310/33—Chemical structure of the base
- C12N2310/331—Universal or degenerate base
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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
- C12N2310/3513—Protein; Peptide
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
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- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Wood Science & Technology (AREA)
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- General Engineering & Computer Science (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
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- Plant Pathology (AREA)
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- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
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- Epidemiology (AREA)
- Communicable Diseases (AREA)
- Cardiology (AREA)
- Urology & Nephrology (AREA)
- Virology (AREA)
- Heart & Thoracic Surgery (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US57404804P | 2004-05-24 | 2004-05-24 | |
| US60/574,048 | 2004-05-24 | ||
| US11/136,245 US20050288246A1 (en) | 2004-05-24 | 2005-05-23 | Peptide conjugated, inosine-substituted antisense oligomer compound and method |
| US11/136,245 | 2005-05-23 | ||
| PCT/US2005/018213 WO2005115479A2 (en) | 2004-05-24 | 2005-05-24 | Peptide conjugated, inosine-substituted antisense oligomer compound and method |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2011130577A Division JP2011172596A (ja) | 2004-05-24 | 2011-06-10 | ペプチドが結合された、イノシン置換アンチセンスオリゴマー化合物および方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2008509701A JP2008509701A (ja) | 2008-04-03 |
| JP2008509701A5 JP2008509701A5 (enExample) | 2008-05-15 |
| JP4974890B2 true JP4974890B2 (ja) | 2012-07-11 |
Family
ID=35451420
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2007527563A Expired - Lifetime JP4974890B2 (ja) | 2004-05-24 | 2005-05-24 | ペプチドが結合された、イノシン置換アンチセンスオリゴマー化合物および方法 |
| JP2011130577A Withdrawn JP2011172596A (ja) | 2004-05-24 | 2011-06-10 | ペプチドが結合された、イノシン置換アンチセンスオリゴマー化合物および方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2011130577A Withdrawn JP2011172596A (ja) | 2004-05-24 | 2011-06-10 | ペプチドが結合された、イノシン置換アンチセンスオリゴマー化合物および方法 |
Country Status (8)
| Country | Link |
|---|---|
| US (3) | US20050288246A1 (enExample) |
| EP (1) | EP1765414B1 (enExample) |
| JP (2) | JP4974890B2 (enExample) |
| AT (1) | ATE529513T1 (enExample) |
| AU (1) | AU2005247460B2 (enExample) |
| CA (1) | CA2565103C (enExample) |
| ES (1) | ES2375631T3 (enExample) |
| WO (1) | WO2005115479A2 (enExample) |
Families Citing this family (46)
| Publication number | Priority date | Publication date | Assignee | Title |
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| EP2351844B1 (en) | 2003-04-29 | 2014-06-11 | Sarepta Therapeutics, Inc. | Compositions for enhancing transport and antisense efficacy of nucleic acid analog into cells |
| US20050288246A1 (en) | 2004-05-24 | 2005-12-29 | Iversen Patrick L | Peptide conjugated, inosine-substituted antisense oligomer compound and method |
| ATE498685T1 (de) | 2004-06-28 | 2011-03-15 | Univ Western Australia | Antisense-oligonukleotide zur induktion von exon- skipping sowie verfahren zur verwendung davon |
| US8067571B2 (en) | 2005-07-13 | 2011-11-29 | Avi Biopharma, Inc. | Antibacterial antisense oligonucleotide and method |
| WO2007009094A2 (en) * | 2005-07-13 | 2007-01-18 | Avi Biopharma, Inc. | Antisense antibacterial method and compound |
| ES2645410T3 (es) * | 2006-05-10 | 2017-12-05 | Sarepta Therapeutics, Inc. | Análogos de oligonucleótidos que tienen enlaces intersubunitarios catiónicos |
| WO2008008476A2 (en) * | 2006-07-12 | 2008-01-17 | The Regents Of The University Of California | Transducible delivery of nucleic acids by reversible phosphotriester charge neutralization protecting groups |
| AU2015200600B2 (en) * | 2007-06-29 | 2017-04-20 | Sarepta Therapeutics, Inc. | Tissue specific peptide conjugates and methods |
| US20100016215A1 (en) | 2007-06-29 | 2010-01-21 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
| CA2703931C (en) | 2007-10-26 | 2016-08-16 | Dynavax Technologies Corporation | Methods and compositions for inhibition of immune responses and autoimmunity |
| PT2203173E (pt) | 2007-10-26 | 2016-03-15 | Academisch Ziekenhuis Leiden | Resumo |
| 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 |
| TR201902952T4 (tr) | 2008-10-24 | 2019-03-21 | Sarepta Therapeutics Inc | Dmd için ekson atlama bileşimleri. |
| NZ595955A (en) | 2009-04-24 | 2012-10-26 | Prosensa Technologies Bv | Oligonucleotide comprising an inosine for treating dmd |
| US20110269665A1 (en) | 2009-06-26 | 2011-11-03 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
| EP3252068B1 (en) | 2009-10-12 | 2025-07-02 | Larry J. Smith | Methods and compositions for modulating gene expression using oligonucleotide based drugs administered in vivo or in vitro |
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| AU2011299233B2 (en) | 2010-09-07 | 2016-09-15 | Integrated Dna Technologies, Inc. | Modifications for antisense compounds |
| WO2012036159A1 (ja) | 2010-09-15 | 2012-03-22 | 日本精工株式会社 | ラジアルころ軸受用保持器 |
| US9161948B2 (en) | 2011-05-05 | 2015-10-20 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| KR102183273B1 (ko) * | 2011-05-05 | 2020-11-27 | 사렙타 쎄러퓨틱스, 인코퍼레이티드 | 펩타이드 올리고뉴클레오타이드 접합체 |
| CN108864192A (zh) | 2011-11-18 | 2018-11-23 | 萨勒普塔医疗公司 | 功能改性的寡核苷酸及其亚单元 |
| NZ627896A (en) | 2012-01-27 | 2016-11-25 | Biomarin Technologies B V | Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy |
| CA2880869A1 (en) | 2012-08-20 | 2014-02-27 | The Regents Of The University Of California | Polynucleotides having bioreversible groups |
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| NZ775701A (en) | 2013-03-14 | 2022-08-26 | Sarepta Therapeutics Inc | Exon skipping compositions for treating muscular dystrophy |
| NZ732507A (en) | 2013-03-15 | 2018-08-31 | Sarepta Therapeutics Inc | Improved compositions for treating muscular dystrophy |
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| JP6901966B2 (ja) | 2014-05-16 | 2021-07-14 | オレゴン ステート ユニバーシティ | アンチセンス抗菌化合物および方法 |
| JP6687542B2 (ja) | 2014-05-19 | 2020-04-22 | オレゴン ステート ユニバーシティ | アンチセンス抗菌化合物および方法 |
| AU2015372560B2 (en) | 2014-12-31 | 2021-12-02 | Board Of Regents, The University Of Texas System | Antisense antibacterial compounds and methods |
| WO2016196670A1 (en) | 2015-06-01 | 2016-12-08 | Sarepta Therapeutics, Inc. | Antisense-induced exon exclusion in type vii collagen |
| US11020417B2 (en) | 2015-06-04 | 2021-06-01 | Sarepta Therapeutics, Inc | Methods and compounds for treatment of lymphocyte-related diseases and conditions |
| US20190030176A1 (en) * | 2015-12-15 | 2019-01-31 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| WO2017112885A1 (en) * | 2015-12-23 | 2017-06-29 | David Greenberg | Antisense antibacterial compounds and methods |
| EP3445405A4 (en) | 2016-04-18 | 2019-12-18 | Sarepta Therapeutics, Inc. | ANTISENSE OLIGOMERS AND METHOD FOR USE THEREOF TO TREAT DISEASES RELATING TO THE ACID ALPHA GLUCOSIDASE GENE |
| KR20190058477A (ko) | 2016-08-17 | 2019-05-29 | 솔스티스 바이올로직스, 리미티드 | 폴리뉴클레오티드 구축물 |
| MD3554553T2 (ro) | 2016-12-19 | 2022-10-31 | Sarepta Therapeutics Inc | Conjugați de oligomeri de omitere a exonului, pentru distrofie musculară |
| WO2019006455A1 (en) | 2017-06-30 | 2019-01-03 | Solstice Biologics, Ltd. | AUXILIARIES OF CHIRAL PHOSPHORAMIDITIS AND METHODS OF USE THEREOF |
| US11555189B2 (en) | 2017-10-18 | 2023-01-17 | Sarepta Therapeutics, Inc. | Antisense oligomer compounds |
| MX2021002745A (es) | 2018-09-11 | 2021-05-12 | Amgen Inc | Metodos de purificacion de oligonucleotidos ricos en guanina. |
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| AU2005247460B2 (en) | 2012-08-02 |
| CA2565103A1 (en) | 2005-12-08 |
| EP1765414A4 (en) | 2009-10-21 |
| WO2005115479A3 (en) | 2007-11-22 |
| HK1101314A1 (en) | 2007-10-12 |
| US20130131312A1 (en) | 2013-05-23 |
| AU2005247460A1 (en) | 2005-12-08 |
| WO2005115479A2 (en) | 2005-12-08 |
| JP2011172596A (ja) | 2011-09-08 |
| EP1765414B1 (en) | 2011-10-19 |
| CA2565103C (en) | 2014-10-14 |
| US20050288246A1 (en) | 2005-12-29 |
| US8877725B2 (en) | 2014-11-04 |
| ATE529513T1 (de) | 2011-11-15 |
| EP1765414A2 (en) | 2007-03-28 |
| US20080182973A1 (en) | 2008-07-31 |
| ES2375631T3 (es) | 2012-03-02 |
| US8053420B2 (en) | 2011-11-08 |
| JP2008509701A (ja) | 2008-04-03 |
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