WO2008018795A1 - Methods and means for treating dna repeat instability associated genetic disorders - Google Patents
Methods and means for treating dna repeat instability associated genetic disorders Download PDFInfo
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- WO2008018795A1 WO2008018795A1 PCT/NL2007/050399 NL2007050399W WO2008018795A1 WO 2008018795 A1 WO2008018795 A1 WO 2008018795A1 NL 2007050399 W NL2007050399 W NL 2007050399W WO 2008018795 A1 WO2008018795 A1 WO 2008018795A1
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- oligonucleotide
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- 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
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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
- A61P25/00—Drugs for disorders of the nervous system
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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- 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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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
- C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
- C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
- C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
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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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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- 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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- 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
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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- C—CHEMISTRY; METALLURGY
- 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
- C12Q2600/00—Oligonucleotides characterized by their use
- C12Q2600/156—Polymorphic or mutational markers
Abstract
Description
Claims
Priority Applications (18)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
JP2009523740A JP6047270B2 (en) | 2006-08-11 | 2007-08-10 | Methods and means for treating genetic disorders associated with instability of DNA repeats |
NZ574807A NZ574807A (en) | 2006-08-11 | 2007-08-10 | Methods and means for treating dna repeat instability associated genetic disorders |
PL07808532T PL2049664T3 (en) | 2006-08-11 | 2007-08-10 | Single stranded oligonucleotides complementary to repetitive elements for treating DNA repeat instability associated genetic disorders |
CA2660523A CA2660523C (en) | 2006-08-11 | 2007-08-10 | Methods and means for treating dna repeat instability associated genetic disorders |
CN2007800298485A CN101501193B (en) | 2006-08-11 | 2007-08-10 | Methods and means for treating DNA repeat instability associated genetic disorders |
US12/377,160 US20100184833A1 (en) | 2006-08-11 | 2007-08-10 | Methods and means for treating dna repeat instability associated genetic disorders |
ES07808532T ES2373246T3 (en) | 2006-08-11 | 2007-08-10 | COMPLEMENTARY MONOCATENARY OLIGONUCLEOTIDES OF REPETITIVE ELEMENTS FOR THE TREATMENT OF GENETIC DISORDERS ASSOCIATED WITH THE INSTABILITY OF DNA REPETITIONS. |
EP07808532A EP2049664B1 (en) | 2006-08-11 | 2007-08-10 | Single stranded oligonucleotides complementary to repetitive elements for treating DNA repeat instability associated genetic disorders |
DK07808532.1T DK2049664T3 (en) | 2006-08-11 | 2007-08-10 | Single-stranded oligonucleotides, complementary to repetitive elements, for the treatment of DNA repetitive instability-associated disorders |
AU2007282224A AU2007282224B2 (en) | 2006-08-11 | 2007-08-10 | Methods and means for treating DNA repeat instability associated genetic disorders |
SI200730778T SI2049664T1 (en) | 2006-08-11 | 2007-08-10 | Single stranded oligonucleotides complementary to repetitive elements for treating DNA repeat instability associated genetic disorders |
AT07808532T ATE524547T1 (en) | 2006-08-11 | 2007-08-10 | SINGLE STRANDED OLIGONUCLEOTIDES COMPLEMENTARY TO REPETITIVE ELEMENTS FOR THE TREATMENT OF DNA REPEATS-INSTABILITY-ASSOCIATED DISEASES |
IL196921A IL196921A (en) | 2006-08-11 | 2009-02-05 | Single stranded oligonucleotides complementary to repetitive elements for treating dna repeat instability associated genetic disorders |
HK09109656.7A HK1130510A1 (en) | 2006-08-11 | 2009-10-20 | Single stranded oligonucleotides complementary to repetitive elements for treating dna repeat instability associated genetic disorders |
US14/809,483 US9890379B2 (en) | 2006-08-11 | 2015-07-27 | Treatment of genetic disorders associated with DNA repeat instability |
US15/855,848 US10689646B2 (en) | 2006-08-11 | 2017-12-27 | Treatment of genetic disorders associated with DNA repeat instability |
US16/660,387 US11274299B2 (en) | 2006-08-11 | 2019-10-22 | Methods and means for treating DNA repeat instability associated genetic disorders |
US17/591,681 US20220267765A1 (en) | 2006-08-11 | 2022-02-03 | Treatment of genetic disorders associated with dna repeat instability |
Applications Claiming Priority (4)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
EP06118809.0 | 2006-08-11 | ||
EP06118809 | 2006-08-11 | ||
EP06119247 | 2006-08-21 | ||
EP06119247.2 | 2006-08-21 |
Related Child Applications (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
US12/377,160 A-371-Of-International US20100184833A1 (en) | 2006-08-11 | 2007-08-10 | Methods and means for treating dna repeat instability associated genetic disorders |
US14/809,483 Division US9890379B2 (en) | 2006-08-11 | 2015-07-27 | Treatment of genetic disorders associated with DNA repeat instability |
Publications (1)
Publication Number | Publication Date |
---|---|
WO2008018795A1 true WO2008018795A1 (en) | 2008-02-14 |
Family
ID=38668759
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PCT/NL2007/050399 WO2008018795A1 (en) | 2006-08-11 | 2007-08-10 | Methods and means for treating dna repeat instability associated genetic disorders |
Country Status (16)
Country | Link |
---|---|
US (5) | US20100184833A1 (en) |
EP (1) | EP2049664B1 (en) |
JP (2) | JP6047270B2 (en) |
AT (1) | ATE524547T1 (en) |
AU (1) | AU2007282224B2 (en) |
CA (1) | CA2660523C (en) |
CY (1) | CY1111975T1 (en) |
DK (1) | DK2049664T3 (en) |
ES (1) | ES2373246T3 (en) |
HK (1) | HK1130510A1 (en) |
IL (1) | IL196921A (en) |
NZ (1) | NZ574807A (en) |
PL (1) | PL2049664T3 (en) |
PT (1) | PT2049664E (en) |
SI (1) | SI2049664T1 (en) |
WO (1) | WO2008018795A1 (en) |
Cited By (48)
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WO2009099326A1 (en) * | 2008-02-08 | 2009-08-13 | Prosensa Holding Bv | Methods and means for treating dna repeat instability associated genetic disorders |
WO2010014592A1 (en) | 2008-07-29 | 2010-02-04 | The Board Of Regents Of The University Of Texas Sytem | Selective inhibition of polyglutamine protein expression |
WO2010115993A1 (en) * | 2009-04-10 | 2010-10-14 | Association Institut De Myologie | Tricyclo-dna antisense oligonucleotides, compositions, and methods for the treatment of disease |
WO2011097641A1 (en) * | 2010-02-08 | 2011-08-11 | Isis Pharmaceuticals, Inc. | Methods and compositions useful in treatment of diseases or conditions related to repeat expansion |
WO2011150408A2 (en) | 2010-05-28 | 2011-12-01 | Avi Biopharma, Inc. | Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups |
WO2012012443A2 (en) | 2010-07-19 | 2012-01-26 | Bennett C Frank | Modulation of dystrophia myotonica-protein kinase (dmpk) expression |
WO2012018257A1 (en) * | 2010-08-05 | 2012-02-09 | Academisch Ziekenhuis Leiden H.O.D.N. Lumc | Antisense oligonucleotide directed removal of proteolytic cleavage sites from proteins |
WO2012138289A1 (en) | 2011-04-08 | 2012-10-11 | Zain-Luqman Rula | Diagnosis and treatment of friedreich's ataxia |
WO2012150960A1 (en) | 2011-05-05 | 2012-11-08 | Avi Biopharma, Inc. | Peptide oligonucleotide conjugates |
US8415465B2 (en) | 2006-01-26 | 2013-04-09 | Isis Pharmaceuticals, Inc. | Compositions and their uses directed to huntingtin |
WO2013142087A1 (en) | 2012-03-20 | 2013-09-26 | Sarepta Therapeutics, Inc. | Boronic acid conjugates of oligonucleotide analogues |
WO2013162363A1 (en) * | 2012-04-23 | 2013-10-31 | Prosensa Technologies B.V. | RNA modulating oligonucleotides with improved characteristics for the treatment of neuromuscular disorders |
US8609065B2 (en) | 2007-07-12 | 2013-12-17 | Prosensa Technologies B.V. | Molecules for targeting compounds to various selected organs, tissues or tumor cells |
US8906873B2 (en) | 2009-09-11 | 2014-12-09 | Isis Pharmaceuticals, Inc. | Modulation of huntingtin expression |
WO2015053624A3 (en) * | 2013-10-07 | 2015-08-27 | Academisch Ziekenhuis Leiden H.O.D.N. Lumc | Antisense oligonucleotide directed removal of proteolytic cleavage sites, the hchwa-d mutation, and trinucleotide repeat expansions |
US9139828B2 (en) | 2008-05-14 | 2015-09-22 | Prosensa Technologies B.V. | Method for efficient exon (44) skipping in duchenne muscular dystrophy and associated means |
US9249243B2 (en) | 2005-07-13 | 2016-02-02 | Sarepta Therapeutics, Inc. | Antibacterial antisense oligonucleotide and method |
US9278987B2 (en) | 2011-11-18 | 2016-03-08 | Sarepta Therapeutics, Inc. | Functionally-modified oligonucleotides and subunits thereof |
WO2016088797A1 (en) * | 2014-12-02 | 2016-06-09 | 国立大学法人東京医科歯科大学 | Nucleic acid reducing als-causing protein toxicity |
US9499818B2 (en) | 2007-10-26 | 2016-11-22 | BioMarin Technologies, B.V. | Methods and means for efficient skipping of at least one of the exons 51-53, 55, 57 and 59 of the human duchenne muscular dystrophy gene |
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US9890379B2 (en) | 2006-08-11 | 2018-02-13 | Biomarin Technologies B.V. | Treatment of genetic disorders associated with DNA repeat instability |
US9944926B2 (en) | 2011-11-30 | 2018-04-17 | Sarepta Therapeutics, Inc. | Induced exon inclusion in spinal muscle atrophy |
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US10041074B2 (en) | 2013-08-16 | 2018-08-07 | Translate Bio Ma, Inc. | Euchromatic region targeting methods for modulating gene expression |
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US10905709B2 (en) | 2015-08-28 | 2021-02-02 | Sarepta Therapeutics, Inc. | Modified antisense oligomers for exon inclusion in spinal muscular atrophy |
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