HRP20200944T1 - Oligonukleotidi koji odgovaraju col7a1 eksonu 73 za terapiju bulozne epidermolize - Google Patents
Oligonukleotidi koji odgovaraju col7a1 eksonu 73 za terapiju bulozne epidermolize Download PDFInfo
- Publication number
- HRP20200944T1 HRP20200944T1 HRP20200944TT HRP20200944T HRP20200944T1 HR P20200944 T1 HRP20200944 T1 HR P20200944T1 HR P20200944T T HRP20200944T T HR P20200944TT HR P20200944 T HRP20200944 T HR P20200944T HR P20200944 T1 HRP20200944 T1 HR P20200944T1
- Authority
- HR
- Croatia
- Prior art keywords
- oligoribonucleotide
- antisense
- mrna
- exon
- therapy
- Prior art date
Links
- 206010014989 Epidermolysis bullosa Diseases 0.000 title 1
- 108091034117 Oligonucleotide Proteins 0.000 title 1
- JLCPHMBAVCMARE-UHFFFAOYSA-N [3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-hydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methyl [5-(6-aminopurin-9-yl)-2-(hydroxymethyl)oxolan-3-yl] hydrogen phosphate Polymers Cc1cn(C2CC(OP(O)(=O)OCC3OC(CC3OP(O)(=O)OCC3OC(CC3O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c3nc(N)[nH]c4=O)C(COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3CO)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cc(C)c(=O)[nH]c3=O)n3cc(C)c(=O)[nH]c3=O)n3ccc(N)nc3=O)n3cc(C)c(=O)[nH]c3=O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)O2)c(=O)[nH]c1=O JLCPHMBAVCMARE-UHFFFAOYSA-N 0.000 title 1
- 238000002560 therapeutic procedure Methods 0.000 title 1
- 108020004519 Antisense Oligoribonucleotides Proteins 0.000 claims 11
- 239000002825 antisense oligoribonucleotide Substances 0.000 claims 11
- 108020004999 messenger RNA Proteins 0.000 claims 6
- 108091027075 5S-rRNA precursor Proteins 0.000 claims 5
- 101000909498 Homo sapiens Collagen alpha-1(VII) chain Proteins 0.000 claims 2
- 102000050118 human COL7A1 Human genes 0.000 claims 2
- 210000004962 mammalian cell Anatomy 0.000 claims 2
- 239000002773 nucleotide Substances 0.000 claims 2
- 125000003729 nucleotide group Chemical group 0.000 claims 2
- 101000587430 Homo sapiens Serine/arginine-rich splicing factor 2 Proteins 0.000 claims 1
- 102100029666 Serine/arginine-rich splicing factor 2 Human genes 0.000 claims 1
- 230000004048 modification Effects 0.000 claims 1
- 238000012986 modification Methods 0.000 claims 1
Classifications
-
- 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
- C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
-
- 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
- A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
-
- 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
- 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
- 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
- C12N15/1138—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 receptors or cell surface proteins
-
- 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
- C12N2310/315—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
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
-
- 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
- C12N2310/3521—Methyl
-
- 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)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Crystallography & Structural Chemistry (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Claims (9)
1. Antisens oligoribonukleotid koji može spriječiti ili smanjiti uključivanje eksona 73 u ljudsku COL7A1 mRNA, kada se navedena mRNA proizvede spajanjem iz pred-mRNA u stanici sisavca, naznačen time što se oligoribonukleotid može žariti na (SRp40 / SC35 koji veže / ESE) element u eksonu 73 karakteriziran prema sekvenci 5'-UUUCCUGG-3 '(SEQ ID NO: 4), pri čemu oligoribonukleotid ima duljinu između 16 i 24 nukleotida.
2. Antisens oligoribonukleotid prema zahtjevu 1, naznačen time što nema više od jedne CpG sekvence.
3. Antisens oligoribonukleotid koji može spriječiti ili smanjiti uključivanje eksona 73 u ljudsku COL7A1 mRNA, kada se navedena mRNA proizvede spajanjem iz pred-mRNA u stanici sisavca, naznačen time što je oligoribonukleotid odabran iz skupine koja se sastoji od antisens oligoribonukleotida (AONi) od SEQ ID NO: 5, 6, 7, 8, 24, 25, 26, 27, 28, 29, 35, 39, 40, 41, 42 i 43.
4. Antisens oligoribonukleotid prema zahtjevu 3, naznačen time što je oligoribonukleotid AON od SEQ ID NO: 35.
5. Antisens oligoribonukleotid prema bilo kojem od zahtjeva 1 do 4, naznačen time što su internukleozidne veze kemijski modificirane.
6. Antisens oligoribonukleotid prema zahtjevu 5, naznačen time što internukleozidna modifikacija veze je fosforotioatna veza.
7. Antisens oligoribonukleotid prema bilo kojem od zahtjeva 1 do 6, naznačen time što su šećerni dijelovi oligoribonukleotida niži 2'-O-alkil.
8. Antisens oligoribonukleotid prema zahtjevu 7, naznačen time što je 2'-O-alkil 2'-O-metil supstituirani šećerni dio.
9. Antisens oligoribonukleotid prema bilo kojem od zahtjeva 1 do 8, naznačen time što je duljina odabrana iz skupine koja se sastoji od 16, 17, 18, 19, 20, 21, 22, 23 ili 24 nukleotida.
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| GBGB1504124.7A GB201504124D0 (en) | 2015-03-11 | 2015-03-11 | Oligonucleotides |
| PCT/EP2016/055360 WO2016142538A1 (en) | 2015-03-11 | 2016-03-11 | Oligonucleotides matching col7a1 exon 73 for epidermolysis bullosa therapy |
| EP16709465.5A EP3268474B1 (en) | 2015-03-11 | 2016-03-11 | Oligonucleotides matching col7a1 exon 73 for epidermolysis bullosa therapy |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| HRP20200944T1 true HRP20200944T1 (hr) | 2020-09-18 |
Family
ID=52998751
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HRP20200944TT HRP20200944T1 (hr) | 2015-03-11 | 2020-06-12 | Oligonukleotidi koji odgovaraju col7a1 eksonu 73 za terapiju bulozne epidermolize |
Country Status (20)
| Country | Link |
|---|---|
| US (2) | US10563198B2 (hr) |
| EP (2) | EP3763816A1 (hr) |
| JP (2) | JP6929783B2 (hr) |
| KR (1) | KR20170120617A (hr) |
| CN (1) | CN107567498B (hr) |
| AU (1) | AU2016231067B2 (hr) |
| BR (1) | BR112017019225A2 (hr) |
| CA (1) | CA2978375A1 (hr) |
| DK (1) | DK3268474T3 (hr) |
| EA (1) | EA035732B1 (hr) |
| ES (1) | ES2803651T3 (hr) |
| GB (1) | GB201504124D0 (hr) |
| HR (1) | HRP20200944T1 (hr) |
| HU (1) | HUE050898T2 (hr) |
| IL (1) | IL254173B (hr) |
| MX (2) | MX2017011599A (hr) |
| PL (1) | PL3268474T3 (hr) |
| PT (1) | PT3268474T (hr) |
| SI (1) | SI3268474T1 (hr) |
| WO (1) | WO2016142538A1 (hr) |
Families Citing this family (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3851531A1 (en) * | 2015-06-01 | 2021-07-21 | Sarepta Therapeutics, Inc. | Antisense-induced exon exclusion in type vii collagen |
| EP3744333A4 (en) * | 2018-01-25 | 2021-11-03 | Osaka University | MEDICAL COMPOSITION FOR THE TREATMENT OF DISEASES ASSOCIATED WITH AN INCREASE IN THE LEVEL OF EXPRESSION OF PERIOSTIN OR A CHANGE IN THE SPLICE VARIANT THEREOF |
| GB201902735D0 (en) | 2019-02-28 | 2019-04-17 | Proqr Therapeutics Ii Bv | Oligonucleotides for use in the treatment of dystrophic epidermolysis bullosa |
| JP2023504314A (ja) | 2019-12-02 | 2023-02-02 | シェイプ セラピューティクス インコーポレイテッド | 治療的編集 |
| US20240110181A1 (en) * | 2020-12-10 | 2024-04-04 | Institut National de la Santé et de la Recherche Médicale | Substances and methods for treating dystrophic epidermolysis bullosa |
| WO2024080311A1 (ja) * | 2022-10-12 | 2024-04-18 | 昌和 栗田 | 遺伝子導入法、遺伝子治療法、及び組織再生法 |
| CN116555349A (zh) * | 2023-01-09 | 2023-08-08 | 中吉智药(南京)生物技术有限公司 | 一种腺相关病毒载体及其构建方法与应用 |
Family Cites Families (35)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
| US7101993B1 (en) | 1990-01-11 | 2006-09-05 | Isis Pharmaceuticals, Inc. | Oligonucleotides containing 2′-O-modified purines |
| US5952221A (en) | 1996-03-06 | 1999-09-14 | Avigen, Inc. | Adeno-associated virus vectors comprising a first and second nucleic acid sequence |
| CA2253724A1 (en) | 1996-04-26 | 1997-11-06 | Case Western Reserve University | Skin regeneration using mesenchymal stem cells |
| JP2002080662A (ja) | 2000-09-08 | 2002-03-19 | Jsr Corp | ゴム組成物 |
| EP1191097A1 (en) | 2000-09-21 | 2002-03-27 | Leids Universitair Medisch Centrum | Induction of exon skipping in eukaryotic cells |
| EP2270024B1 (en) | 2001-06-21 | 2018-10-24 | Ionis Pharmaceuticals, Inc. | Antisense modulation of superoxide dismutase 1, soluble expression |
| AU2002366799A1 (en) | 2001-12-19 | 2003-07-09 | Alwyn Company, Inc. | Allantoin-containing preparations for administration as gels and aerosols |
| US20040096833A1 (en) | 2002-11-16 | 2004-05-20 | Isis Pharmaceuticals Inc. | Modulation of FBP-interacting repressor expression |
| US20040092464A1 (en) | 2002-11-11 | 2004-05-13 | Isis Pharmaceuticals Inc. | Modulation of mitogen-activated protein kinase kinase kinase 11 expression |
| DE10238298A1 (de) | 2002-08-21 | 2004-03-04 | Beiersdorf Ag | Verwendung von Antisense-Oligonucleotiden zur Behandlung von degenerativen Hauterscheinungen |
| 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 |
| US8765169B2 (en) | 2004-02-13 | 2014-07-01 | Smith & Nephew, Inc. | Wound healing profile |
| US20080207538A1 (en) | 2004-03-11 | 2008-08-28 | Lawrence David S | Enhanced Production of Functional Proteins From Defective Genes |
| USRE47769E1 (en) | 2004-06-28 | 2019-12-17 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| GB0505202D0 (en) | 2005-03-14 | 2005-04-20 | Intercytex Ltd | Skin equivalent culture |
| WO2007069813A1 (en) | 2005-12-16 | 2007-06-21 | Modern Cell & Tissue Technologies Inc. | Methods for culturing mesenchymal stem cell and compositions comprising mesenchymal stem cell for reparing skin defects |
| PL2161038T3 (pl) | 2006-01-26 | 2014-06-30 | Ionis Pharmaceuticals Inc | Kompozycje i ich zastosowanie ukierunkowane na huntingtynę |
| EP1857548A1 (en) * | 2006-05-19 | 2007-11-21 | Academisch Ziekenhuis Leiden | Means and method for inducing exon-skipping |
| US20100184833A1 (en) | 2006-08-11 | 2010-07-22 | Prosenta Technologies B.V. | Methods and means for treating dna repeat instability associated genetic disorders |
| EP2134848A2 (en) | 2007-03-07 | 2009-12-23 | Aarhus Universitet | Pig model for breast cancer, mitochondria related protein folding disorders and/or epidermolysis bullosa simplex |
| US8077297B2 (en) | 2008-06-30 | 2011-12-13 | Nellcor Puritan Bennett Ireland | Methods and systems for discriminating bands in scalograms |
| EP2151248A1 (en) | 2008-07-30 | 2010-02-10 | Johann Bauer | Improved pre-mRNA trans-splicing molecule (RTM) molecules and their uses |
| US9611471B2 (en) | 2010-08-05 | 2017-04-04 | Academisch Ziekenhuis Leiden | Antisense oligonucleotide directed removal of proteolytic cleavage sites from proteins |
| CN103501793A (zh) * | 2011-02-08 | 2014-01-08 | 夏洛特-梅克伦堡医院(商业用名:卡罗来纳保健系统) | 反义寡核苷酸 |
| WO2013033436A1 (en) * | 2011-09-01 | 2013-03-07 | University Of Iowa Research Foundation | Oligonucleotide-based probes for detection of bacterial nucleases |
| AU2013201303C1 (en) * | 2011-10-06 | 2016-06-23 | MiRagen Therapeutics, Inc. | Control of whole body energy homeostasis by microRNA regulation |
| WO2013053819A1 (en) * | 2011-10-11 | 2013-04-18 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Exon skipping therapy for dystrophic epidermolysis bullosa |
| KR20150009521A (ko) | 2012-03-07 | 2015-01-26 | 파이브로셀 테크놀로지스, 인코퍼레이티드 | 국소피부제제 및 개인맞춤형 피부치료방법 |
| EP2850184A4 (en) | 2012-05-16 | 2016-01-27 | Rana Therapeutics Inc | COMPOSITIONS AND METHOD FOR MODULATING GENE EXPRESSION |
| WO2015004133A1 (en) | 2013-07-08 | 2015-01-15 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods for performing antisense oligonucleotide-mediated exon skipping in the retina of a subject in need thereof |
| WO2016081296A2 (en) | 2014-11-17 | 2016-05-26 | Rev Pharma Corp | Topical medicament for skin and mucosal injuries associated with epidermolisis bullosa |
| EP3851531A1 (en) | 2015-06-01 | 2021-07-21 | Sarepta Therapeutics, Inc. | Antisense-induced exon exclusion in type vii collagen |
| LT3307277T (lt) | 2015-06-15 | 2020-12-28 | Tirmed Pharma Ab | Viengrandžiai oligonukleotidai, skirti naudoti mediciniškai gydant odos sutrikimus |
| WO2017078526A2 (en) | 2015-11-05 | 2017-05-11 | Rijksuniversiteit Groningen | Antisense oligonucleotide-mediated exon skipping as systemic therapy for recessive dystrophic epidermolysis bullosa (rdeb). |
-
2015
- 2015-03-11 GB GBGB1504124.7A patent/GB201504124D0/en not_active Ceased
-
2016
- 2016-03-11 DK DK16709465.5T patent/DK3268474T3/da active
- 2016-03-11 KR KR1020177025038A patent/KR20170120617A/ko active IP Right Grant
- 2016-03-11 HU HUE16709465A patent/HUE050898T2/hu unknown
- 2016-03-11 ES ES16709465T patent/ES2803651T3/es active Active
- 2016-03-11 EA EA201791986A patent/EA035732B1/ru unknown
- 2016-03-11 PT PT167094655T patent/PT3268474T/pt unknown
- 2016-03-11 JP JP2017547468A patent/JP6929783B2/ja active Active
- 2016-03-11 EP EP20175344.9A patent/EP3763816A1/en active Pending
- 2016-03-11 WO PCT/EP2016/055360 patent/WO2016142538A1/en active Application Filing
- 2016-03-11 PL PL16709465T patent/PL3268474T3/pl unknown
- 2016-03-11 MX MX2017011599A patent/MX2017011599A/es unknown
- 2016-03-11 EP EP16709465.5A patent/EP3268474B1/en active Active
- 2016-03-11 CA CA2978375A patent/CA2978375A1/en active Pending
- 2016-03-11 US US15/554,420 patent/US10563198B2/en active Active
- 2016-03-11 SI SI201630794T patent/SI3268474T1/sl unknown
- 2016-03-11 BR BR112017019225-0A patent/BR112017019225A2/pt not_active Application Discontinuation
- 2016-03-11 AU AU2016231067A patent/AU2016231067B2/en active Active
- 2016-03-11 CN CN201680015142.2A patent/CN107567498B/zh active Active
-
2017
- 2017-08-27 IL IL254173A patent/IL254173B/en unknown
- 2017-09-08 MX MX2021009135A patent/MX2021009135A/es unknown
-
2020
- 2020-01-17 US US16/746,403 patent/US11352626B2/en active Active
- 2020-06-12 HR HRP20200944TT patent/HRP20200944T1/hr unknown
-
2021
- 2021-08-11 JP JP2021131294A patent/JP7188810B2/ja active Active
Also Published As
| Publication number | Publication date |
|---|---|
| JP6929783B2 (ja) | 2021-09-01 |
| CN107567498A (zh) | 2018-01-09 |
| JP7188810B2 (ja) | 2022-12-13 |
| AU2016231067B2 (en) | 2022-04-21 |
| IL254173A0 (en) | 2017-10-31 |
| ES2803651T3 (es) | 2021-01-28 |
| HUE050898T2 (hu) | 2021-01-28 |
| US10563198B2 (en) | 2020-02-18 |
| IL254173B (en) | 2022-03-01 |
| JP2022000020A (ja) | 2022-01-04 |
| PT3268474T (pt) | 2020-06-17 |
| AU2016231067A1 (en) | 2017-10-26 |
| SI3268474T1 (sl) | 2020-07-31 |
| KR20170120617A (ko) | 2017-10-31 |
| MX2021009135A (es) | 2021-09-10 |
| EP3268474A1 (en) | 2018-01-17 |
| DK3268474T3 (da) | 2020-06-15 |
| JP2018509903A (ja) | 2018-04-12 |
| NZ736083A (en) | 2021-08-27 |
| PL3268474T3 (pl) | 2020-08-24 |
| US11352626B2 (en) | 2022-06-07 |
| WO2016142538A1 (en) | 2016-09-15 |
| EA201791986A1 (ru) | 2018-01-31 |
| CA2978375A1 (en) | 2016-09-15 |
| MX2017011599A (es) | 2018-06-15 |
| US20200399638A1 (en) | 2020-12-24 |
| US20180216106A1 (en) | 2018-08-02 |
| CN107567498B (zh) | 2021-09-21 |
| EA035732B1 (ru) | 2020-07-31 |
| BR112017019225A2 (pt) | 2018-05-08 |
| EP3763816A1 (en) | 2021-01-13 |
| GB201504124D0 (en) | 2015-04-22 |
| EP3268474B1 (en) | 2020-05-27 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| HRP20200944T1 (hr) | Oligonukleotidi koji odgovaraju col7a1 eksonu 73 za terapiju bulozne epidermolize | |
| HRP20190992T1 (hr) | Suprimiranje gena za huntingtin inducirano s rnai | |
| HRP20210612T1 (hr) | Postupci i pripravci za specifičnu inhibiciju glikolat oksidaze (hao1) pomoću dvolančane rnk | |
| JP2018513668A5 (hr) | ||
| HRP20210227T1 (hr) | Oligonukleotidi za indukciju ekspresije paternalnog gena ube3a | |
| JP2016523564A5 (hr) | ||
| JP2018509903A5 (hr) | ||
| JP2016522674A5 (hr) | ||
| NZ733882A (en) | Compositions for modulating c9orf72 expression | |
| JP2017079776A5 (hr) | ||
| NZ593618A (en) | Gnaq targeted dsrna compositions and methods for inhibiting expression | |
| JP2015523855A5 (hr) | ||
| MX2017011013A (es) | Terapia de oligonucleotidos para amaurosis congenita de leber. | |
| JP2015519057A5 (hr) | ||
| NZ592867A (en) | Compositions and methods for inhibiting expression of transthyretin | |
| JP2015518711A5 (hr) | ||
| NZ709013A (en) | Pcsk9 irna compositions and methods of use thereof | |
| MX359400B (es) | Reduccion de metal pesado en plantas. | |
| HRP20221498T1 (hr) | Pripravci serpinc1 irnk i postupci njihove upotrebe | |
| WO2009078685A3 (en) | Novel sirna structure for minimizing off-target effects and relaxing saturation of rnai machinery and the use thereof | |
| JP2016521556A5 (hr) | ||
| WO2012178033A3 (en) | Serpina1 sirnas: compositions of matter and methods of treatment | |
| WO2011078797A3 (en) | Antisense oligonucleotides and uses threreof | |
| NZ630914A (en) | Methods of treating diabetes and/or promoting survival of pancreatic islets after transplantation | |
| EP2896696A3 (en) | Modifications for antisense compounds |