HRP20190387T1 - Pripravci koji preskaču ekson za dmd - Google Patents
Pripravci koji preskaču ekson za dmd Download PDFInfo
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- HRP20190387T1 HRP20190387T1 HRP20190387TT HRP20190387T HRP20190387T1 HR P20190387 T1 HRP20190387 T1 HR P20190387T1 HR P20190387T T HRP20190387T T HR P20190387TT HR P20190387 T HRP20190387 T HR P20190387T HR P20190387 T1 HRP20190387 T1 HR P20190387T1
- Authority
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- Croatia
- Prior art keywords
- antisense oligonucleotide
- pharmaceutically acceptable
- acceptable salt
- salt according
- oligonucleotide
- Prior art date
Links
- 239000000203 mixture Substances 0.000 title 1
- 108091034117 Oligonucleotide Proteins 0.000 claims 18
- 239000000074 antisense oligonucleotide Substances 0.000 claims 15
- 238000012230 antisense oligonucleotides Methods 0.000 claims 15
- 150000003839 salts Chemical class 0.000 claims 11
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 claims 4
- ISAKRJDGNUQOIC-UHFFFAOYSA-N Uracil Chemical compound O=C1C=CNC(=O)N1 ISAKRJDGNUQOIC-UHFFFAOYSA-N 0.000 claims 4
- RWQNBRDOKXIBIV-UHFFFAOYSA-N thymine Chemical compound CC1=CNC(=O)NC1=O RWQNBRDOKXIBIV-UHFFFAOYSA-N 0.000 claims 4
- 201000006938 muscular dystrophy Diseases 0.000 claims 3
- 239000000825 pharmaceutical preparation Substances 0.000 claims 3
- 201000006935 Becker muscular dystrophy Diseases 0.000 claims 2
- 239000002202 Polyethylene glycol Substances 0.000 claims 2
- 125000003827 glycol group Chemical group 0.000 claims 2
- 125000004573 morpholin-4-yl group Chemical group N1(CCOCC1)* 0.000 claims 2
- 125000004193 piperazinyl group Chemical group 0.000 claims 2
- 229920001223 polyethylene glycol Polymers 0.000 claims 2
- 229940113082 thymine Drugs 0.000 claims 2
- ZIBGPFATKBEMQZ-UHFFFAOYSA-N triethylene glycol Chemical group OCCOCCOCCO ZIBGPFATKBEMQZ-UHFFFAOYSA-N 0.000 claims 2
- 229940035893 uracil Drugs 0.000 claims 2
- MHCMWGPPLOSCJY-UHFFFAOYSA-N 4-$l^{1}-azanylmorpholine Chemical compound [N]N1CCOCC1 MHCMWGPPLOSCJY-UHFFFAOYSA-N 0.000 claims 1
- LRSASMSXMSNRBT-UHFFFAOYSA-N 5-methylcytosine Chemical compound CC1=CNC(=O)N=C1N LRSASMSXMSNRBT-UHFFFAOYSA-N 0.000 claims 1
- OKTJSMMVPCPJKN-UHFFFAOYSA-N Carbon Chemical compound [C] OKTJSMMVPCPJKN-UHFFFAOYSA-N 0.000 claims 1
- 101001053946 Homo sapiens Dystrophin Proteins 0.000 claims 1
- OAICVXFJPJFONN-UHFFFAOYSA-N Phosphorus Chemical compound [P] OAICVXFJPJFONN-UHFFFAOYSA-N 0.000 claims 1
- 229910052799 carbon Inorganic materials 0.000 claims 1
- 230000000295 complement effect Effects 0.000 claims 1
- 239000003085 diluting agent Substances 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 108020004999 messenger RNA Proteins 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 229910052698 phosphorus Inorganic materials 0.000 claims 1
- 239000011574 phosphorus Substances 0.000 claims 1
Classifications
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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
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- 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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- 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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- 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
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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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- 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
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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
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3233—Morpholino-type ring
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/334—Modified C
- C12N2310/3341—5-Methylcytosine
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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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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
- C12N2310/3533—Halogen
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- C12N2320/30—Special therapeutic applications
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/33—Alteration of splicing
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- C12Q2527/00—Reactions demanding special reaction conditions
- C12Q2527/107—Temperature of melting, i.e. Tm
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- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Physics & Mathematics (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Epidemiology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
- Peptides Or Proteins (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Claims (12)
1. Protusmisleni oligonukleotid koji sadrži 20-35 morfolino podjedinica povezanih s vezama među podjedinicama koje sadrže fosfor koje spajaju morfolino dušik jedne podjedinice sa 5' 'egzocikličkim ugljikom susjedne podjedinice, koji sadrži baznu sekvencu SEQ ID NO: 382, ili sekvencu koja ima s njom barem 80% identičnosti sekvence, pri čemu su timinske baze proizvoljno uracilne baze, te pri čemu je protusmisleni oligonukleotid komplementaran s ljudskim distrofinom pred-mRNA da se inducira preskakanje eksona 52; ili njegova farmaceutski prihvatljiva sol.
2. Protusmisleni oligonukleotid ili njegova farmaceutski prihvatljiva sol prema zahtjevu 1, naznačen time što protusmisleni oligonukleotid ili njegova farmaceutski prihvatljiva sol sadrži 20-25 morfolino podjedinica.
3. Protusmisleni oligonukleotid ili njegova farmaceutski prihvatljiva sol prema zahtjevu 1 ili zahtjevu 2, naznačen time što ima uracilne baze (U) na mjestu timinskih baza (T).
4. Protusmisleni oligonukleotid ili njegova farmaceutski prihvatljiva sol prema bilo kojem od zahtjeva 1-3, naznačen time što sadrži bazu 5-metilcitozina.
5. Protusmisleni oligonukleotid ili njegova farmaceutski prihvatljiva sol prema zahtjevima 1-4, naznačen time što je oligonukleotid konjugiran na lanac polietilen glikola.
6. Protusmisleni oligonukleotid ili njegova farmaceutski prihvatljiva sol prema zahtjevima 1-4, naznačen time što je oligonukleotid konjugiran na dio trietilenglikola.
7. Protusmisleni oligonukleotid ili njegova farmaceutski prihvatljiva sol prema zahtjevima 1-4, naznačen time što je oligonukleotid konjugiran na lanac polietilen glikola vezan preko supstituiranog dijela piperazinila.
8. Protusmisleni oligonukleotid ili njegova farmaceutski prihvatljiva sol prema zahtjevima 1-4 naznačen time što je protusmisleni oligonukleotid konjugiran na dio trietilenglikola vezan preko supstituiranog dijela piperazinila.
9. Farmaceutski pripravak naznačen time što sadrži protusmisleni oligonukleotid ili njegovu farmaceutski prihvatljivu sol prema bilo kojem od prethodnih zahtjeva i farmaceutski prihvatljiv nosač i/ili razrjeđivač.
10. Protusmisleni oligonukleotid ili njegova farmaceutski prihvatljiva sol prema bilo kojem od zahtjeva 1 do 8, ili farmaceutski pripravak prema zahtjevu 9 naznačen time što se koristi u liječenju mišićne distrofije.
11. Protusmisleni oligonukleotid ili farmaceutski pripravak za uporabu prema zahtjevu 10, naznačen time što je mišićna distrofija Duchenneova mišićna distrofija (DMD).
12. Protusmisleni oligonukleotid ili farmaceutski pripravak za uporabu prema zahtjevu 10, naznačen time što je mišićna distrofija Beckerova mišićna distrofija (BMD).
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US10841608P | 2008-10-24 | 2008-10-24 | |
EP16172354.9A EP3133160B1 (en) | 2008-10-24 | 2009-10-23 | Exon skipping compositions for dmd |
Publications (1)
Publication Number | Publication Date |
---|---|
HRP20190387T1 true HRP20190387T1 (hr) | 2019-05-31 |
Family
ID=41510869
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
HRP20190387TT HRP20190387T1 (hr) | 2008-10-24 | 2019-02-28 | Pripravci koji preskaču ekson za dmd |
Country Status (22)
Country | Link |
---|---|
US (14) | US8871918B2 (hr) |
EP (7) | EP3133160B1 (hr) |
JP (10) | JP5864257B2 (hr) |
KR (5) | KR20180011364A (hr) |
CN (3) | CN105779453A (hr) |
AU (1) | AU2009308167B2 (hr) |
BR (3) | BRPI0920276B1 (hr) |
CA (2) | CA2740328A1 (hr) |
CY (1) | CY1122369T1 (hr) |
DK (1) | DK3133160T3 (hr) |
ES (2) | ES2714787T3 (hr) |
HK (1) | HK1200868A1 (hr) |
HR (1) | HRP20190387T1 (hr) |
HU (1) | HUE042979T2 (hr) |
IL (5) | IL304042A (hr) |
LT (1) | LT3133160T (hr) |
PL (1) | PL3133160T3 (hr) |
PT (1) | PT3133160T (hr) |
SI (1) | SI3133160T1 (hr) |
TR (1) | TR201902952T4 (hr) |
TW (6) | TWI488965B (hr) |
WO (1) | WO2010048586A1 (hr) |
Families Citing this family (119)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
CA2524255C (en) * | 2003-03-21 | 2014-02-11 | Academisch Ziekenhuis Leiden | Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure |
ATE479763T1 (de) | 2003-04-29 | 2010-09-15 | Avi Biopharma Inc | Zusammensetzungen zur verbesserung der antisense- wirksamkeit und des transports von nukleinsäureanalog in zellen |
CA2553104A1 (en) | 2004-01-23 | 2005-08-11 | Avi Biopharma, Inc. | Antisense oligomers and methods for inducing immune tolerance and immunosuppression |
US20050288246A1 (en) | 2004-05-24 | 2005-12-29 | Iversen Patrick L | Peptide conjugated, inosine-substituted antisense oligomer compound and method |
USRE48960E1 (en) | 2004-06-28 | 2022-03-08 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
EP4272748A3 (en) | 2004-06-28 | 2024-03-27 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
EP1855694B1 (en) | 2005-02-09 | 2020-12-02 | Sarepta Therapeutics, Inc. | Antisense composition for treating muscle atrophy |
US8067571B2 (en) | 2005-07-13 | 2011-11-29 | Avi Biopharma, Inc. | Antibacterial antisense oligonucleotide and method |
US20100016215A1 (en) * | 2007-06-29 | 2010-01-21 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
AU2008271050B2 (en) * | 2007-06-29 | 2014-11-06 | Sarepta Therapeutics, Inc. | Tissue specific peptide conjugates and methods |
DK2203173T3 (en) | 2007-10-26 | 2016-02-29 | Academisch Ziekenhuis Leiden | Materials and methods for prevention of muscle diseases |
WO2009086469A2 (en) | 2007-12-28 | 2009-07-09 | Avi Biopharma, Inc. | Immunomodulatory agents and methods of use |
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 |
ES2714787T3 (es) | 2008-10-24 | 2019-05-30 | Sarepta Therapeutics Inc | Composiciones de omisión exónica para DMD |
PL2607484T3 (pl) * | 2008-10-27 | 2016-06-30 | Biomarin Tech Bv | Sposoby i środki do wydajnego pomijania eksonu 45 w pre-mRNA dystrofii mięśniowej Duchenne'a |
ES2593836T3 (es) * | 2009-04-24 | 2016-12-13 | Biomarin Technologies B.V. | Oligonucleótido que comprende una inosina para tratar la DMD |
US20110269665A1 (en) | 2009-06-26 | 2011-11-03 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
IL297299A (en) * | 2009-11-12 | 2022-12-01 | Univ Western Australia | Antisense oligonucleotides for inducing exon skipping in the dystrophin gene |
TWI616454B (zh) | 2010-05-28 | 2018-03-01 | 薩羅塔治療公司 | 具有經修飾之單元間連結及/或末端基團之寡核苷酸類似物 |
TWI541024B (zh) | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
WO2012031243A2 (en) | 2010-09-03 | 2012-03-08 | Avi Biopharma, Inc. | dsRNA MOLECULES COMPRISING OLIGONUCLEOTIDE ANALOGS HAVING MODIFIED INTERSUBUNIT LINKAGES AND/OR TERMINAL GROUPS |
JP2014507143A (ja) | 2011-02-08 | 2014-03-27 | ザ シャーロット−メクレンバーグ ホスピタル オーソリティ ドゥーイング/ビジネス/アズ キャロライナズ ヘルスケア システム | アンチセンスオリゴヌクレオチド |
US9161948B2 (en) | 2011-05-05 | 2015-10-20 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
WO2012150960A1 (en) * | 2011-05-05 | 2012-11-08 | Avi Biopharma, Inc. | Peptide oligonucleotide conjugates |
US20140080896A1 (en) * | 2011-08-30 | 2014-03-20 | The Regents Of The University Of California | Identification of small molecules that facilitate therapeutic exon skipping |
US20130085139A1 (en) | 2011-10-04 | 2013-04-04 | Royal Holloway And Bedford New College | Oligomers |
KR20140097398A (ko) * | 2011-11-18 | 2014-08-06 | 사렙타 쎄러퓨틱스, 인코퍼레이티드 | 기능적으로-변형된 올리고뉴클레오티드 및 이의 서브유니트 |
PL2788487T3 (pl) | 2011-12-08 | 2018-10-31 | Sarepta Therapeutics, Inc. | Analogi oligonukleotydów ukierunkowane na ludzki LMNA |
US9512424B2 (en) * | 2011-12-28 | 2016-12-06 | Nippon Shinyaku Co., Ltd. | Antisense nucleic acids |
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 |
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 |
EP2812313A4 (en) | 2012-02-09 | 2015-09-30 | Novus Int Inc | FUNCTIONALIZED POLYMERIC COMPOSITIONS |
KR102079284B1 (ko) | 2012-03-20 | 2020-02-19 | 사렙타 쎄러퓨틱스, 인코퍼레이티드 | 올리고뉴클레오티드 유사체의 보론산 접합체 |
DE102012103041A1 (de) | 2012-04-10 | 2013-10-10 | Eberhard-Karls-Universität Tübingen Universitätsklinikum | Behandlung der dilativen Kardiomyopathie mittels Antisense-Oligonucleotiden |
EP2841572B1 (en) | 2012-04-27 | 2019-06-19 | Duke University | Genetic correction of mutated genes |
CA2877644A1 (en) * | 2012-07-03 | 2014-01-09 | Prosensa Technologies B.V. | Oligonucleotide for the treatment of muscular dystrophy patients |
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