CY1116305T1 - Μεθοδοι και μεσα για την αποτελεσματικη παραλειψη του εξονιου 45 εις το προ-mrna της μυϊκης δυστροφιας τυπου duchenne - Google Patents
Μεθοδοι και μεσα για την αποτελεσματικη παραλειψη του εξονιου 45 εις το προ-mrna της μυϊκης δυστροφιας τυπου duchenneInfo
- Publication number
- CY1116305T1 CY1116305T1 CY20151100228T CY151100228T CY1116305T1 CY 1116305 T1 CY1116305 T1 CY 1116305T1 CY 20151100228 T CY20151100228 T CY 20151100228T CY 151100228 T CY151100228 T CY 151100228T CY 1116305 T1 CY1116305 T1 CY 1116305T1
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- instruments
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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
- 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
- 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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- 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
- A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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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
- C12N2310/11—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
- C12N2310/30—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
- C12N2310/321—2'-O-R Modification
Landscapes
- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Neurology (AREA)
- General Health & Medical Sciences (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Pharmacology & Pharmacy (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Neurosurgery (AREA)
- Biotechnology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Psychiatry (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Hospice & Palliative Care (AREA)
- Biochemistry (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
Abstract
Η εφεύρεση αφορά μέθοδο, όπου χρησιμοποιείται μόριο για την επαγωγή και/ή προώθηση της παράλειψης τουλάχιστον ενός από τα εξόνιο 43, εξόνιο 46, εξόνια 50-53 του προ-mRNA DMD εις ασθενή, κατά προτίμηση εις απομονωμένο κύτταρο ασθενή, όπου η μέθοδος περιλαμβάνει παροχή μορίου εις το εν λόγω κύτταρο και/ή τον εν λόγω ασθενή. Η εφεύρεση αφορά επίσης το εν λόγω μόριο καθαυτό.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
PCT/NL2008/050673 WO2009054725A2 (en) | 2007-10-26 | 2008-10-27 | Means and methods for counteracting muscle disorders |
EP09788160.1A EP2344637B2 (en) | 2008-10-27 | 2009-01-13 | Methods and means for efficient skipping of exon 45 in duchenne muscular dystrophy pre-mrna |
Publications (1)
Publication Number | Publication Date |
---|---|
CY1116305T1 true CY1116305T1 (el) | 2017-02-08 |
Family
ID=40873329
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CY20151100228T CY1116305T1 (el) | 2008-10-27 | 2015-03-04 | Μεθοδοι και μεσα για την αποτελεσματικη παραλειψη του εξονιου 45 εις το προ-mrna της μυϊκης δυστροφιας τυπου duchenne |
Country Status (15)
Country | Link |
---|---|
EP (4) | EP2607484B1 (el) |
JP (2) | JP5786109B2 (el) |
AU (1) | AU2009310557B2 (el) |
CA (3) | CA2741629C (el) |
CY (1) | CY1116305T1 (el) |
DK (2) | DK2607484T3 (el) |
ES (2) | ES2562658T3 (el) |
HK (1) | HK1160169A1 (el) |
HR (1) | HRP20160078T1 (el) |
IL (2) | IL212509A (el) |
NZ (1) | NZ592498A (el) |
PL (1) | PL2607484T3 (el) |
PT (2) | PT2344637E (el) |
SI (1) | SI2607484T1 (el) |
WO (2) | WO2010050801A1 (el) |
Families Citing this family (51)
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USRE48960E1 (en) | 2004-06-28 | 2022-03-08 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
ATE498685T1 (de) | 2004-06-28 | 2011-03-15 | Univ Western Australia | Antisense-oligonukleotide zur induktion von exon- skipping sowie verfahren zur verwendung davon |
DK2203173T3 (en) | 2007-10-26 | 2016-02-29 | Academisch Ziekenhuis Leiden | Materials and methods for prevention of muscle diseases |
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 |
CN102203253B (zh) | 2008-10-24 | 2016-04-06 | 萨雷普塔治疗公司 | 用于dmd的多外显子跳跃组合物 |
US8802437B2 (en) * | 2009-09-24 | 2014-08-12 | Cellectis | Meganuclease reagents of uses thereof for treating genetic diseases caused by frame shift/non sense mutations |
KR102113306B1 (ko) | 2009-11-12 | 2020-05-21 | 더 유니버시티 오브 웨스턴 오스트레일리아 | 안티센스 분자 및 이를 이용한 질환 치료방법 |
TWI541024B (zh) | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
BR112013020273A2 (pt) * | 2011-02-08 | 2016-10-18 | Charlotte Mecklenburg Hospital | oligonucleotídeos antissenso |
RU2619184C2 (ru) * | 2011-12-28 | 2017-05-12 | Ниппон Синяку Ко., Лтд. | Антисмысловые нуклеиновые кислоты |
CN104203289B (zh) | 2012-01-27 | 2020-11-03 | 比奥马林技术公司 | 用于治疗杜兴型肌营养不良症和贝克型肌营养不良症的具有改善特性的rna调节性寡核苷酸 |
EP3608407A1 (en) * | 2012-07-03 | 2020-02-12 | BioMarin Technologies B.V. | Oligonucleotide for the treatment of muscular dystrophy patients |
EA035882B1 (ru) | 2013-03-14 | 2020-08-27 | Сарепта Терапьютикс, Инк. | Антисмысловые олигонуклеотиды, обеспечивающие пропуск экзонов, для лечения мышечной дистрофии |
WO2014144978A2 (en) | 2013-03-15 | 2014-09-18 | Sarepta Therapeutics, Inc. | Improved compositions for treating muscular dystrophy |
TWI736514B (zh) * | 2013-09-05 | 2021-08-21 | 美商薩羅塔治療公司 | 酸性α葡萄糖苷酶之反義股誘導之外顯子2包含 |
JP6519842B2 (ja) * | 2013-10-04 | 2019-05-29 | 国立大学法人神戸大学 | 福山型筋ジストロフィー治療用アンチセンス核酸 |
MY193708A (en) * | 2014-03-12 | 2022-10-26 | Nippon Shinyaku Co Ltd | Antisense nucleic acids |
HRP20220379T1 (hr) * | 2014-06-10 | 2022-05-27 | Erasmus University Medical Center Rotterdam | Antisense oligonukleotidi korisni u liječenju pompeove bolesti |
PT3351633T (pt) * | 2015-09-15 | 2020-07-29 | Nippon Shinyaku Co Ltd | Ácido nucleico antissentido |
PE20181353A1 (es) | 2015-10-09 | 2018-08-22 | Wave Life Sciences Ltd | Composiciones oligonucleotidicas y sus metodos |
US11339392B2 (en) | 2016-03-02 | 2022-05-24 | The Board Of Trustees Of The Leland Stanford Junior University | Pan-genotypic agents against influenza virus and methods of using the same |
LT3800257T (lt) * | 2016-03-02 | 2024-07-25 | The Board Of Trustees Of The Leland Stanford Junior University | Pangenotipiniai agentai nuo gripo virusų ir jų naudojimo būdai |
MA45328A (fr) | 2016-04-01 | 2019-02-06 | Avidity Biosciences Llc | Compositions acide nucléique-polypeptide et utilisations de celles-ci |
US11060089B2 (en) | 2016-04-18 | 2021-07-13 | Sarepta Therapeutics, Inc. | Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene |
MA45290A (fr) * | 2016-05-04 | 2019-03-13 | Wave Life Sciences Ltd | Procédés et compositions d'agents biologiquement actifs |
KR20190024977A (ko) | 2016-06-30 | 2019-03-08 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이상증에 대한 엑손 스킵핑 올리고머 |
MX2018016253A (es) * | 2016-07-05 | 2019-09-09 | Biomarin Tech Bv | Oligonucleotidos de conmutacion o modulacion de empalme de pre-arnm que comprenden radicales de andamio biciclicos, con caracteristicas mejoradas para el tratamiento de trastornos geneticos. |
PL3554554T3 (pl) | 2016-12-19 | 2022-12-19 | Sarepta Therapeutics, Inc. | Koniugaty oligomerów z pominięciem egzonu w dystrofii mięśniowej |
CA3047010A1 (en) | 2016-12-19 | 2018-06-28 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
KR20230110811A (ko) | 2016-12-19 | 2023-07-25 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체 |
SG10202107429WA (en) | 2017-01-06 | 2021-08-30 | Avidity Biosciences Inc | Nucleic acid-polypeptide compositions and methods of inducing exon skipping |
GB201711809D0 (en) * | 2017-07-21 | 2017-09-06 | Governors Of The Univ Of Alberta | Antisense oligonucleotide |
EA201991450A1 (ru) | 2017-09-22 | 2019-12-30 | Сарепта Терапьютикс, Инк. | Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии |
US20210145852A1 (en) | 2017-09-28 | 2021-05-20 | Sarepta Therapeutics, Inc. | Combination Therapies for Treating Muscular Dystrophy |
SG11202003126VA (en) | 2017-10-04 | 2020-05-28 | Avidity Biosciences Inc | Nucleic acid-polypeptide compositions and uses thereof |
KR102443358B1 (ko) | 2017-12-06 | 2022-09-14 | 어비디티 바이오사이언시스 인크. | 근위축증 및 근긴장성 이영양증을 치료하는 조성물 및 방법 |
US10765760B2 (en) | 2018-05-29 | 2020-09-08 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
WO2019241385A2 (en) * | 2018-06-13 | 2019-12-19 | Sarepta Therapeutics, Inc. | Exon skipping oligomers for muscular dystropy |
JP2021533200A (ja) | 2018-08-02 | 2021-12-02 | ダイン セラピューティクス, インコーポレーテッドDyne Therapeutics, Inc. | 顔面・肩甲・上腕筋ジストロフィーを処置するための筋標的化複合体およびそれらの使用 |
US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
SG11202100934PA (en) | 2018-08-02 | 2021-02-25 | Dyne Therapeutics Inc | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
WO2020118246A1 (en) * | 2018-12-06 | 2020-06-11 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
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EP4079329A4 (en) * | 2019-12-19 | 2024-04-17 | Nippon Shinyaku Co., Ltd. | ANTISENSE NUCLEIC ACID ENABLING EXON SKIPPING |
CN118109468A (zh) | 2019-12-26 | 2024-05-31 | 日本新药株式会社 | 诱导外显子50的跳读的反义核酸 |
IL295967A (en) | 2020-02-28 | 2022-10-01 | Nippon Shinyaku Co Ltd | Antisense nucleic acids for splicing on exon 51 |
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JP2003511362A (ja) | 1999-10-04 | 2003-03-25 | ユニバーシティー オブ メディシン アンド デンティストリー オブ ニュー ジャージー | 新規カルバメートおよび尿素類 |
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JP4836366B2 (ja) * | 2000-08-25 | 2011-12-14 | 雅文 松尾 | デュシェンヌ型筋ジストロフィー治療剤 |
WO2004083432A1 (en) * | 2003-03-21 | 2004-09-30 | Academisch Ziekenhuis Leiden | Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure |
ATE498685T1 (de) * | 2004-06-28 | 2011-03-15 | Univ Western Australia | Antisense-oligonukleotide zur induktion von exon- skipping sowie verfahren zur verwendung davon |
WO2006112705A2 (en) * | 2005-04-22 | 2006-10-26 | Academisch Ziekenhuis Leiden | Modulation of exon recognition in pre-mrna by interfering with the binding of sr proteins and by interfering with secondary rna structure. |
EP1857548A1 (en) * | 2006-05-19 | 2007-11-21 | Academisch Ziekenhuis Leiden | Means and method for inducing exon-skipping |
DK2203173T3 (en) | 2007-10-26 | 2016-02-29 | Academisch Ziekenhuis Leiden | Materials and methods for prevention of muscle diseases |
US8084601B2 (en) | 2008-09-11 | 2011-12-27 | Royal Holloway And Bedford New College Royal Holloway, University Of London | Oligomers |
CN102203253B (zh) * | 2008-10-24 | 2016-04-06 | 萨雷普塔治疗公司 | 用于dmd的多外显子跳跃组合物 |
US9607308B2 (en) | 2011-06-29 | 2017-03-28 | American Express Travel Related Services Company, Inc. | Spend based digital ad targeting and measurement |
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2009
- 2009-01-13 EP EP13160338.3A patent/EP2607484B1/en not_active Revoked
- 2009-01-13 AU AU2009310557A patent/AU2009310557B2/en active Active
- 2009-01-13 JP JP2011533126A patent/JP5786109B2/ja active Active
- 2009-01-13 CA CA2741629A patent/CA2741629C/en active Active
- 2009-01-13 EP EP09788160.1A patent/EP2344637B2/en active Active
- 2009-01-13 PT PT09788160T patent/PT2344637E/pt unknown
- 2009-01-13 ES ES13160338.3T patent/ES2562658T3/es active Active
- 2009-01-13 WO PCT/NL2009/050006 patent/WO2010050801A1/en active Application Filing
- 2009-01-13 DK DK13160338.3T patent/DK2607484T3/en active
- 2009-01-13 SI SI200931381A patent/SI2607484T1/sl unknown
- 2009-01-13 ES ES09788160.1T patent/ES2532634T5/es active Active
- 2009-01-13 PL PL13160338T patent/PL2607484T3/pl unknown
- 2009-01-13 PT PT131603383T patent/PT2607484E/pt unknown
- 2009-01-13 DK DK09788160.1T patent/DK2344637T4/en active
- 2009-03-11 EP EP18180952.6A patent/EP3400948B1/en active Active
- 2009-03-11 EP EP09788170.0A patent/EP2349287B1/en active Active
- 2009-03-11 CA CA3017539A patent/CA3017539A1/en active Pending
- 2009-03-11 JP JP2011533127A patent/JP5905260B2/ja active Active
- 2009-03-11 WO PCT/NL2009/050113 patent/WO2010050802A2/en active Application Filing
- 2009-03-11 NZ NZ592498A patent/NZ592498A/en unknown
- 2009-03-11 CA CA2741793A patent/CA2741793C/en active Active
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2011
- 2011-04-27 IL IL212509A patent/IL212509A/en active IP Right Grant
- 2011-04-27 IL IL212508A patent/IL212508A/en active IP Right Grant
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2012
- 2012-01-16 HK HK12100495.6A patent/HK1160169A1/xx active IP Right Maintenance
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2015
- 2015-03-04 CY CY20151100228T patent/CY1116305T1/el unknown
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2016
- 2016-01-25 HR HRP20160078TT patent/HRP20160078T1/hr unknown
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