PL2548560T3 - Kompozycje i sposoby modulowania splicingu SMN2 - Google Patents
Kompozycje i sposoby modulowania splicingu SMN2Info
- Publication number
- PL2548560T3 PL2548560T3 PL12188625T PL12188625T PL2548560T3 PL 2548560 T3 PL2548560 T3 PL 2548560T3 PL 12188625 T PL12188625 T PL 12188625T PL 12188625 T PL12188625 T PL 12188625T PL 2548560 T3 PL2548560 T3 PL 2548560T3
- Authority
- PL
- Poland
- Prior art keywords
- modulation
- compositions
- methods
- smn2 splicing
- smn2
- Prior art date
Links
- 101000617738 Homo sapiens Survival motor neuron protein Proteins 0.000 title 1
- 102100021947 Survival motor neuron protein Human genes 0.000 title 1
- 239000000203 mixture Substances 0.000 title 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
-
- 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
- C07H21/02—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 ribosyl as saccharide radical
-
- 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
- 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
- C12N2310/32—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
- C12N2310/33—Chemical structure of the base
- C12N2310/334—Modified C
- C12N2310/3341—5-Methylcytosine
-
- 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)
- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (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)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US69354205P | 2005-06-23 | 2005-06-23 | |
| EP12188625.3A EP2548560B1 (en) | 2005-06-23 | 2006-06-23 | Compositions and methods for modulation of SMN2 splicing |
| EP06773838A EP1910395B1 (en) | 2005-06-23 | 2006-06-23 | Compositions and methods for modulation of SMN2 splicing |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PL2548560T3 true PL2548560T3 (pl) | 2015-11-30 |
Family
ID=37595872
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PL12188625T PL2548560T3 (pl) | 2005-06-23 | 2006-06-23 | Kompozycje i sposoby modulowania splicingu SMN2 |
| PL06773838T PL1910395T3 (pl) | 2005-06-23 | 2006-06-23 | Kompozycja i sposób modulacji splicingu SMN2 |
| PL17185196T PL3308788T3 (pl) | 2005-06-23 | 2006-06-23 | Kompozycje i sposoby modulacji splicingu smn2 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PL06773838T PL1910395T3 (pl) | 2005-06-23 | 2006-06-23 | Kompozycja i sposób modulacji splicingu SMN2 |
| PL17185196T PL3308788T3 (pl) | 2005-06-23 | 2006-06-23 | Kompozycje i sposoby modulacji splicingu smn2 |
Country Status (13)
| Country | Link |
|---|---|
| US (6) | US8361977B2 (pl) |
| EP (5) | EP2548560B1 (pl) |
| CY (2) | CY2017040I1 (pl) |
| DK (3) | DK2548560T3 (pl) |
| ES (3) | ES2545223T3 (pl) |
| HK (1) | HK1247103B (pl) |
| HU (2) | HUE027486T2 (pl) |
| LT (2) | LT3308788T (pl) |
| LU (1) | LUC00046I2 (pl) |
| PL (3) | PL2548560T3 (pl) |
| PT (3) | PT1910395E (pl) |
| SI (2) | SI2548560T1 (pl) |
| WO (1) | WO2007002390A2 (pl) |
Families Citing this family (102)
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| US8361977B2 (en) | 2005-06-23 | 2013-01-29 | Isis Pharmaceuticals, Inc. | Compositions and methods for modulation of SMN2 splicing |
| US8067571B2 (en) | 2005-07-13 | 2011-11-29 | Avi Biopharma, Inc. | Antibacterial antisense oligonucleotide and method |
| DK2203173T3 (en) * | 2007-10-26 | 2016-02-29 | Academisch Ziekenhuis Leiden | Materials and methods for prevention of muscle diseases |
| US8637478B2 (en) | 2007-11-13 | 2014-01-28 | Isis Pharmaceuticals, Inc. | Compounds and methods for modulating protein expression |
| US8633019B2 (en) | 2008-05-27 | 2014-01-21 | Ptc Therapeutics, Inc. | Methods for treating spinal muscular atrophy |
| WO2010019243A1 (en) | 2008-08-13 | 2010-02-18 | Ptc Therapeutics, Inc. | Methods for treating viral infections |
| US9394333B2 (en) | 2008-12-02 | 2016-07-19 | Wave Life Sciences Japan | Method for the synthesis of phosphorus atom modified nucleic acids |
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