SI2612917T1 - Protismiselna nukleinska kislina - Google Patents
Protismiselna nukleinska kislinaInfo
- Publication number
- SI2612917T1 SI2612917T1 SI201130767A SI201130767A SI2612917T1 SI 2612917 T1 SI2612917 T1 SI 2612917T1 SI 201130767 A SI201130767 A SI 201130767A SI 201130767 A SI201130767 A SI 201130767A SI 2612917 T1 SI2612917 T1 SI 2612917T1
- Authority
- SI
- Slovenia
- Prior art keywords
- nucleic acid
- antisense nucleic
- antisense
- acid
- nucleic
- Prior art date
Links
- 230000000692 anti-sense effect Effects 0.000 title 1
- 150000007523 nucleic acids Chemical class 0.000 title 1
- 102000039446 nucleic acids Human genes 0.000 title 1
- 108020004707 nucleic acids Proteins 0.000 title 1
Classifications
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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
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
- C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4707—Muscular dystrophy
- C07K14/4708—Duchenne dystrophy
-
- 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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
-
- 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
-
- 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
-
- 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
- C12N2310/314—Phosphoramidates
- C12N2310/3145—Phosphoramidates with the nitrogen in 3' or 5'-position
-
- 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
-
- 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
-
- 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
-
- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/33—Alteration of splicing
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Biochemistry (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Epidemiology (AREA)
- Toxicology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Physical Education & Sports Medicine (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Neurology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Saccharide Compounds (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2010196032 | 2010-09-01 | ||
| EP11821996.3A EP2612917B1 (en) | 2010-09-01 | 2011-08-31 | Antisense nucleic acid |
| PCT/JP2011/070318 WO2012029986A1 (ja) | 2010-09-01 | 2011-08-31 | アンチセンス核酸 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| SI2612917T1 true SI2612917T1 (sl) | 2016-05-31 |
Family
ID=45773054
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| SI201130767A SI2612917T1 (sl) | 2010-09-01 | 2011-08-31 | Protismiselna nukleinska kislina |
| SI201131801T SI3018211T1 (sl) | 2010-09-01 | 2011-08-31 | Protismiselne nukleinske kisline |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| SI201131801T SI3018211T1 (sl) | 2010-09-01 | 2011-08-31 | Protismiselne nukleinske kisline |
Country Status (21)
| Country | Link |
|---|---|
| US (15) | US9079934B2 (sl) |
| EP (6) | EP4403632A3 (sl) |
| JP (11) | JP5363655B2 (sl) |
| KR (1) | KR101310569B1 (sl) |
| CN (1) | CN103154245B (sl) |
| AU (1) | AU2011296882B2 (sl) |
| CA (1) | CA2809637C (sl) |
| CY (2) | CY1117367T1 (sl) |
| DK (2) | DK2612917T3 (sl) |
| ES (2) | ES2567411T3 (sl) |
| HR (2) | HRP20160336T1 (sl) |
| HU (2) | HUE046364T2 (sl) |
| LT (1) | LT3018211T (sl) |
| PL (2) | PL2612917T3 (sl) |
| PT (1) | PT3018211T (sl) |
| RS (2) | RS54649B1 (sl) |
| RU (1) | RU2567664C2 (sl) |
| SI (2) | SI2612917T1 (sl) |
| SM (1) | SMT201600111B (sl) |
| TW (1) | TWI541024B (sl) |
| WO (1) | WO2012029986A1 (sl) |
Families Citing this family (62)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| USRE48960E1 (en) | 2004-06-28 | 2022-03-08 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| EP2206781B1 (en) | 2004-06-28 | 2015-12-02 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| WO2006086667A2 (en) | 2005-02-09 | 2006-08-17 | Avi Bio Pharma, Inc. | Antisense composition and method for treating muscle atrophy |
| CN101896186A (zh) | 2007-10-26 | 2010-11-24 | 莱顿教学医院 | 对抗肌肉病症的方式和方法 |
| 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 |
| US8084601B2 (en) | 2008-09-11 | 2011-12-27 | Royal Holloway And Bedford New College Royal Holloway, University Of London | Oligomers |
| SI3133160T1 (sl) | 2008-10-24 | 2019-05-31 | Sarepta Therapeutics, Inc. | Sestavki, ki preskakujejo ekson za DMD |
| ES2693459T3 (es) | 2009-11-12 | 2018-12-11 | The University Of Western Australia | Moléculas antisentido y métodos para el tratamiento de patologías |
| TWI541024B (zh) * | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
| US20130085139A1 (en) | 2011-10-04 | 2013-04-04 | Royal Holloway And Bedford New College | Oligomers |
| EP4043039A1 (en) | 2012-01-27 | 2022-08-17 | BioMarin Technologies B.V. | Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy |
| EP3885439A1 (en) * | 2012-12-20 | 2021-09-29 | Sarepta Therapeutics, Inc. | Improved exon skipping compositions for treating muscular dystrophy |
| MX366274B (es) * | 2013-03-14 | 2019-07-04 | Sarepta Therapeutics Inc | Composiciones para el salto del exón para el tratamiento de distrofia muscular. |
| EP3633035A1 (en) | 2013-03-14 | 2020-04-08 | Sarepta Therapeutics, Inc. | Exon skipping compositions for treating muscular dystrophy |
| BR112015022998A2 (pt) | 2013-03-15 | 2017-11-14 | Sarepta Therapeutics Inc | composições melhoradas para o tratamento de distrofia muscular |
| TR201901939T4 (tr) * | 2014-03-12 | 2019-03-21 | Nat Center Neurology & Psychiatry | Antisens nükleik asit. |
| EP3146970B1 (en) * | 2014-05-19 | 2022-11-02 | KNC Laboratories Co., Ltd. | Nucleic acid drug for inducing skipping of variant exon of cd44 gene and increasing expression of normal type cd44 mrna |
| MX2016016526A (es) * | 2014-06-17 | 2017-04-04 | Nippon Shinyaku Co Ltd | Acidos nucleicos antisentido. |
| MA41795A (fr) | 2015-03-18 | 2018-01-23 | Sarepta Therapeutics Inc | Exclusion d'un exon induite par des composés antisens dans la myostatine |
| WO2017047707A1 (ja) | 2015-09-15 | 2017-03-23 | 日本新薬株式会社 | アンチセンス核酸 |
| US10563199B2 (en) * | 2015-09-16 | 2020-02-18 | Nippon Shinyaku Co., Ltd. | Antisense nucleic acid for treating amyotrophy |
| AU2016334232B2 (en) | 2015-10-09 | 2022-05-26 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
| CN108699555A (zh) | 2015-10-09 | 2018-10-23 | 萨勒普塔医疗公司 | 用于治疗杜兴肌营养不良和相关病症的组合物和方法 |
| FR3044926B1 (fr) | 2015-12-09 | 2020-01-31 | Genethon | Outils de therapie genique efficaces pour le saut de l'exon 53 de la dystrophine |
| KR101686379B1 (ko) | 2016-02-05 | 2016-12-13 | 박정열 | 휴대용 구이기 |
| FI3464306T3 (fi) | 2016-05-24 | 2024-05-16 | Sarepta Therapeutics Inc | Menetelmiä fosforodiamidaattimorfolino-oligomeerien valmistamiseksi |
| US11472824B2 (en) | 2016-05-24 | 2022-10-18 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
| MA45362A (fr) | 2016-05-24 | 2019-04-10 | Sarepta Therapeutics Inc | Procédés de préparation d'oligomères morpholino de phosphorodiamidate |
| SG10202101830WA (en) * | 2016-05-24 | 2021-04-29 | Sarepta Therapeutics Inc | Processes for preparing oligomers |
| WO2018005805A1 (en) | 2016-06-30 | 2018-01-04 | Sarepta Therapeutics, Inc. | Exon skipping oligomers for muscular dystrophy |
| JP7022079B2 (ja) * | 2016-06-30 | 2022-02-17 | サレプタ セラピューティクス， インコーポレイテッド | ホスホロジアミダートモルホリノオリゴマーを調製するためのプロセス |
| KR101715572B1 (ko) | 2016-10-11 | 2017-03-22 | 박종찬 | 구이대 승강구조를 갖는 양방향 구이기 |
| DK3554553T3 (da) | 2016-12-19 | 2022-09-19 | Sarepta Therapeutics Inc | Exon-overspringnings-oligomerkonjugat til muskeldystrofi |
| PL3554552T3 (pl) | 2016-12-19 | 2022-11-21 | Sarepta Therapeutics, Inc. | Koniugaty oligomerów do pomijania egzonów dla dystrofii mięśniowej |
| MX2019006989A (es) | 2016-12-19 | 2019-08-16 | Sarepta Therapeutics Inc | Conjugados de oligomeros de omision de exon para distrofia muscular. |
| WO2018223056A1 (en) | 2017-06-02 | 2018-12-06 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| US20230038956A1 (en) * | 2017-08-31 | 2023-02-09 | Sarepta Therapeutics, Inc. | Methods for treating muscular dystrophy |
| EA201991450A1 (ru) | 2017-09-22 | 2019-12-30 | Сарепта Терапьютикс, Инк. | Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии |
| WO2019067979A1 (en) | 2017-09-28 | 2019-04-04 | Sarepta Therapeutics, Inc. | POLYTHERAPIES FOR TREATING MUSCLE DYSTROPHY |
| JP2021521794A (ja) * | 2018-04-26 | 2021-08-30 | サレプタ セラピューティクス， インコーポレイテッド | 筋ジストロフィーに対するエクソンスキッピングオリゴマーおよびオリゴマーコンジュゲート |
| EP3790596A4 (en) * | 2018-05-11 | 2022-04-06 | Wave Life Sciences Ltd. | OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF USE |
| US10765760B2 (en) | 2018-05-29 | 2020-09-08 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
| US20210261963A1 (en) | 2018-06-26 | 2021-08-26 | Nippon Shinyaku Co., Ltd. | Composition comprising antisense oligonucleotide and use thereof for treatment of duchenne muscular dystrophy |
| EP3830259A4 (en) | 2018-08-02 | 2022-05-04 | Dyne Therapeutics, Inc. | MUSCLE TARGETING COMPLEXES AND THEIR USES FOR THE TREATMENT OF FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY |
| EP3829595A4 (en) | 2018-08-02 | 2022-08-24 | Dyne Therapeutics, Inc. | MUSCLE TARGETING COMPLEXES AND THEIR USES FOR THE TREATMENT OF DYSTROPHINOPATHIES |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| JP7185239B2 (ja) * | 2018-12-25 | 2022-12-07 | 国立研究開発法人国立精神・神経医療研究センター | 随時尿中細胞を用いた筋系細胞の誘導方法 |
| GB201821269D0 (en) * | 2018-12-28 | 2019-02-13 | Nippon Shinyaku Co Ltd | Myostatin signal inhibitor |
| CA3165961A1 (en) | 2019-12-26 | 2021-07-01 | Nippon Shinyaku Co., Ltd. | Antisense nucleic acid that induces skipping of exon 50 |
| US20230140736A1 (en) | 2020-02-28 | 2023-05-04 | Nippon Shinyaku Co., Ltd. | Antisense nucleic acid inducing skipping of exon 51 |
| WO2021182517A1 (ja) | 2020-03-11 | 2021-09-16 | 隆光 矢野 | －１フレームシフトを誘導するための１本鎖核酸分子及び組成物 |
| CA3211038A1 (en) | 2021-02-12 | 2022-08-18 | Oxford University Innovation Limited | Cell-penetrating peptide conjugates and methods of their use |
| WO2022232478A1 (en) | 2021-04-30 | 2022-11-03 | Sarepta Therapeutics, Inc. | Treatment methods for muscular dystrophy |
| US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| WO2023026994A1 (ja) | 2021-08-21 | 2023-03-02 | 武田薬品工業株式会社 | ヒトトランスフェリンレセプター結合ペプチド－薬物コンジュゲート |
| CA3229962A1 (en) | 2021-08-24 | 2023-03-02 | Peptidream Inc. | Human transferrin receptor-binding antibody-peptide conjugate |
| WO2023127918A1 (ja) | 2021-12-27 | 2023-07-06 | 日本新薬株式会社 | オリゴ核酸化合物の製造方法 |
| EP4215614A1 (en) | 2022-01-24 | 2023-07-26 | Dynacure | Combination therapy for dystrophin-related diseases |
| WO2023168427A1 (en) | 2022-03-03 | 2023-09-07 | Yale University | Compositions and methods for delivering therapeutic polynucleotides for exon skipping |
| WO2023171820A1 (ja) * | 2022-03-11 | 2023-09-14 | 日本新薬株式会社 | キャリアペプチドが連結された核酸 |
| IL315280A (en) | 2022-03-17 | 2024-10-01 | Sarepta Therapeutics Inc | Morpholino phosphorodiamidate oligomer conjugates |
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| JP2924179B2 (ja) | 1993-02-19 | 1999-07-26 | 日本新薬株式会社 | グリセロール誘導体，デバイス及び医薬組成物 |
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| JP2000325085A (ja) | 1999-05-21 | 2000-11-28 | Masafumi Matsuo | デュシェンヌ型筋ジストロフィー治療剤 |
| US6653467B1 (en) | 2000-04-26 | 2003-11-25 | Jcr Pharmaceutical Co., Ltd. | Medicament for treatment of Duchenne muscular dystrophy |
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| US6727355B2 (en) | 2000-08-25 | 2004-04-27 | Jcr Pharmaceuticals Co., Ltd. | Pharmaceutical composition for treatment of Duchenne muscular dystrophy |
| 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 |
| EP1191097A1 (en) | 2000-09-21 | 2002-03-27 | Leids Universitair Medisch Centrum | Induction of exon skipping in eukaryotic cells |
| ITRM20020253A1 (it) | 2002-05-08 | 2003-11-10 | Univ Roma | Molecole chimeriche di snrna con sequenze antisenso per le giunzioni di splicing del gene della distrofina e applicazioni terapeutiche. |
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