LUC00046I1 - - Google Patents
Info
- Publication number
- LUC00046I1 LUC00046I1 LU00046C LUC00046C LUC00046I1 LU C00046 I1 LUC00046 I1 LU C00046I1 LU 00046 C LU00046 C LU 00046C LU C00046 C LUC00046 C LU C00046C LU C00046 I1 LUC00046 I1 LU C00046I1
- Authority
- LU
- Luxembourg
Links
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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- 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
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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
-
- 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 (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US69354205P | 2005-06-23 | 2005-06-23 | |
EP06773838A EP1910395B1 (fr) | 2005-06-23 | 2006-06-23 | Compositions et procédés de modulation de l épissage de SMN2 |
Publications (2)
Publication Number | Publication Date |
---|---|
LUC00046I1 true LUC00046I1 (fr) | 2017-11-10 |
LUC00046I2 LUC00046I2 (fr) | 2018-01-10 |
Family
ID=37595872
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
LU00046C LUC00046I2 (fr) | 2005-06-23 | 2017-11-07 |
Country Status (13)
Country | Link |
---|---|
US (6) | US8361977B2 (fr) |
EP (5) | EP1910395B1 (fr) |
CY (2) | CY2017040I2 (fr) |
DK (3) | DK2548560T3 (fr) |
ES (3) | ES2702531T3 (fr) |
HK (2) | HK1181307A1 (fr) |
HU (2) | HUE027486T2 (fr) |
LT (2) | LT3308788T (fr) |
LU (1) | LUC00046I2 (fr) |
PL (3) | PL2548560T3 (fr) |
PT (3) | PT1910395E (fr) |
SI (2) | SI3308788T1 (fr) |
WO (1) | WO2007002390A2 (fr) |
Families Citing this family (97)
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US7838657B2 (en) | 2004-12-03 | 2010-11-23 | University Of Massachusetts | Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences |
AU2006213686A1 (en) | 2005-02-09 | 2006-08-17 | Avi Bio Pharma, Inc. | Antisense composition and method for treating muscle atrophy |
ES2702531T3 (es) | 2005-06-23 | 2019-03-01 | Biogen Ma Inc | Composiciones y procedimientos para modulación de splicing de SMN2 |
US8067571B2 (en) | 2005-07-13 | 2011-11-29 | Avi Biopharma, Inc. | Antibacterial antisense oligonucleotide and method |
AU2008317566B2 (en) * | 2007-10-26 | 2014-05-01 | Academisch Ziekenhuis Leiden | Means and methods for counteracting muscle disorders |
EP2219680A2 (fr) | 2007-11-13 | 2010-08-25 | Isis Pharmaceuticals, Inc. | Composés et méthodes destinées à la modulation de l'expression de protéines |
WO2009151546A2 (fr) * | 2008-05-27 | 2009-12-17 | Ptc Therapeutics, Inc. | Procédés de traitement d'une atrophie musculaire spinale |
US8932818B2 (en) | 2008-08-13 | 2015-01-13 | Ptc Therapeutics, Inc. | Screening assays for compounds that modulate programmed ribosomal frameshifting |
EP2370451B1 (fr) | 2008-12-02 | 2016-11-16 | Wave Life Sciences Japan, Inc. | Procédé pour la synthèse d'acides nucléiques modifiés au niveau de l'atome de phosphore |
IT1398361B1 (it) * | 2009-01-21 | 2013-02-22 | Fond Santa Lucia | Uso di mutanti dominanti negativi di sam68 per il trattamento della sma |
PT2417257E (pt) * | 2009-04-10 | 2016-06-03 | Universität Bern | Oligonucleótidos triciclo-dna anti-sentido, composições, e métodos para o tratamento de doença |
US20120149757A1 (en) * | 2009-04-13 | 2012-06-14 | Krainer Adrian R | Compositions and methods for modulation of smn2 splicing |
MX2011014004A (es) * | 2009-06-17 | 2012-06-19 | Isis Pharmaceuticals Inc | Composiciones y metodos para la modulacion de division de smn2 en un sujeto. |
RU2612521C2 (ru) | 2009-07-06 | 2017-03-09 | Онтории, Инк. | Новые пролекарства нуклеиновых кислот и способы их применения |
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EP2576574A2 (fr) | 2010-05-28 | 2013-04-10 | Sarepta Therapeutics, Inc. | Analogues oligonucléotidiques ayant des liaisons modifiées entre sous-unités et/ou des groupes terminaux modifiés |
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TWI541024B (zh) | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
WO2012031243A2 (fr) | 2010-09-03 | 2012-03-08 | Avi Biopharma, Inc. | Molécules d'arnds comportant des analogues oligonucléotidiques ayant des liaisons entre sous-unités et/ou des groupes terminaux modifiés |
WO2012039448A1 (fr) | 2010-09-24 | 2012-03-29 | 株式会社キラルジェン | Groupe auxiliaire asymétrique |
IT1405793B1 (it) * | 2010-10-15 | 2014-01-24 | Univ Ferrara | Molecola di u1snrna umano modificato, gene codificante per la molecola di u1snrna umano modificato, vettore di espressione includente il gene, e loro uso in terapia genica |
EP2638163B1 (fr) | 2010-11-12 | 2017-05-17 | The General Hospital Corporation | Arn non codants associés à polycomb |
US9920317B2 (en) | 2010-11-12 | 2018-03-20 | The General Hospital Corporation | Polycomb-associated non-coding RNAs |
US20140128449A1 (en) * | 2011-04-07 | 2014-05-08 | The Board Of Regents Of The University Of Texas System | Oligonucleotide modulation of splicing |
IL273838B (en) | 2011-05-05 | 2022-09-01 | Sarepta Therapeutics Inc | Peptide and oligonucleotide conjugates, preparations containing them and their uses |
WO2012178122A2 (fr) * | 2011-06-23 | 2012-12-27 | Cold Spring Harbor Laboratory | Modèle phénocopique de maladie |
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US20130085139A1 (en) | 2011-10-04 | 2013-04-04 | Royal Holloway And Bedford New College | Oligomers |
CA2855241A1 (fr) | 2011-11-11 | 2013-05-16 | Santaris Pharma A/S | Composes pour la modulation de l'epissage de smn2 |
MX354940B (es) | 2011-11-18 | 2018-03-27 | Sarepta Therapeutics Inc | Oligonucleotidos funcionalmente modificados y subunidades de los mismos. |
US9944926B2 (en) | 2011-11-30 | 2018-04-17 | Sarepta Therapeutics, Inc. | Induced exon inclusion in spinal muscle atrophy |
EP3858847A1 (fr) | 2011-11-30 | 2021-08-04 | Sarepta Therapeutics, Inc. | Oligonucléotides pour le traitement de maladies à répétition nucléotidique étendue |
WO2013086207A1 (fr) * | 2011-12-06 | 2013-06-13 | The Ohio State University | Agents de contraste non ioniques de faible osmolarité pour l'administration d'oligonucléotides antisens et le traitement de maladie |
BR112014018427B1 (pt) | 2012-01-27 | 2021-11-03 | Biomarin Technologies B.V. | Oligonucleotídeos moduladores de rna com características melhoradas para o tratamento da distrofia muscular de duchenne e becker |
US10837014B2 (en) | 2012-05-16 | 2020-11-17 | Translate Bio Ma, Inc. | Compositions and methods for modulating SMN gene family expression |
CA2873797A1 (fr) | 2012-05-16 | 2013-11-21 | Rana Therapeutics Inc. | Compositions et methodes pour moduler l'expression de utrn |
CN104540947A (zh) | 2012-05-16 | 2015-04-22 | Rana医疗有限公司 | 用于调节smn基因家族表达的组合物和方法 |
CA2873779A1 (fr) | 2012-05-16 | 2013-11-21 | Rana Therapeutics Inc. | Compositions et methodes pour moduler l'expression de mecp2 |
CA2879023C (fr) | 2012-07-13 | 2017-03-28 | Wave Life Sciences Japan | Groupe auxiliaire asymetrique |
CN104661664B (zh) | 2012-07-13 | 2020-07-03 | 波涛生命科学有限公司 | 手性控制 |
EP2943225A4 (fr) * | 2013-01-09 | 2016-07-13 | Ionis Pharmaceuticals Inc | Compositions et procédés pour la modulation de l'épissage de smn2 chez un sujet |
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WO2015120450A1 (fr) * | 2014-02-10 | 2015-08-13 | Ohio State Innovation Foundation | Oligonucléotides antisens dans le traitement de l'amyotrophie spinale |
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CA2966044A1 (fr) | 2014-10-30 | 2016-05-06 | The General Hospital Corporation | Procedes de modulation de la repression genique dependant d'atrx |
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