ATE409224T2 - INDUKTION VON ßEXON-SKIPPINGß IN EUKARYOTISCHEN ZELLEN - Google Patents

INDUKTION VON ßEXON-SKIPPINGß IN EUKARYOTISCHEN ZELLEN Download PDF

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Publication number
ATE409224T2
ATE409224T2 AT05076770T AT05076770T ATE409224T2 AT E409224 T2 ATE409224 T2 AT E409224T2 AT 05076770 T AT05076770 T AT 05076770T AT 05076770 T AT05076770 T AT 05076770T AT E409224 T2 ATE409224 T2 AT E409224T2
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Prior art keywords
exon
cell
skipping
instance
ßexon
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AT05076770T
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English (en)
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Ommen Garrit-Jan Boudewijn Van
Deutekom Judith Christina Van
Dunnen Johannes Theodorus Den
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Academisch Ziekenhuis Leiden
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    • 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
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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
    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/04—Drugs for skeletal disorders for non-specific disorders of the connective tissue
    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A61P35/04—Antineoplastic agents specific for metastasis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00—Drugs for disorders of the endocrine system
    • A61P5/14—Drugs for disorders of the endocrine system of the thyroid hormones, e.g. T3, T4
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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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    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • 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
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    • 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
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2310/32—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
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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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule

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  • Health & Medical Sciences (AREA)
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  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
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  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Oncology (AREA)
  • Neurology (AREA)
  • Diabetes (AREA)
  • Dispersion Chemistry (AREA)
  • Neurosurgery (AREA)
  • Hematology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Communicable Diseases (AREA)
  • Endocrinology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
AT05076770T 2000-09-21 2001-09-21 INDUKTION VON ßEXON-SKIPPINGß IN EUKARYOTISCHEN ZELLEN ATE409224T2 (de)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
EP00203283A EP1191097A1 (de) 2000-09-21 2000-09-21 Induktion von "exon-skipping" in eukaryotischen Zellen

Publications (1)

Publication Number Publication Date
ATE409224T2 true ATE409224T2 (de) 2008-10-15

Family

ID=8172043

Family Applications (1)

Application Number Title Priority Date Filing Date
AT05076770T ATE409224T2 (de) 2000-09-21 2001-09-21 INDUKTION VON ßEXON-SKIPPINGß IN EUKARYOTISCHEN ZELLEN

Country Status (16)

Country Link
US (6) US7973015B2 (de)
EP (14) EP1191097A1 (de)
JP (7) JP4846965B2 (de)
AT (1) ATE409224T2 (de)
AU (4) AU2002211062C1 (de)
CA (1) CA2423044C (de)
CY (10) CY1109601T1 (de)
DE (1) DE60135936D1 (de)
DK (10) DK2594642T3 (de)
ES (11) ES2581285T3 (de)
LT (3) LT2940139T (de)
NZ (1) NZ524853A (de)
PT (7) PT2940139T (de)
SI (1) SI2801618T1 (de)
TR (1) TR201810606T4 (de)
WO (1) WO2002024906A1 (de)

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EP2636740B1 (de) 2016-11-09
EP1619249A1 (de) 2006-01-25
DK2284264T3 (en) 2017-03-13
DK2636741T3 (en) 2016-07-04
DK2940139T3 (en) 2018-10-08
DK1619249T4 (da) 2020-04-06
US20150322434A1 (en) 2015-11-12
SI2801618T1 (sl) 2017-07-31
US20090228998A1 (en) 2009-09-10
LT2801618T (lt) 2017-09-11
EP2602322B1 (de) 2016-03-02
JP2016104795A (ja) 2016-06-09
EP2636741A1 (de) 2013-09-11
AU1106202A (en) 2002-04-02
EP2594641B1 (de) 2015-12-30
AU2002211062B2 (en) 2007-11-29
WO2002024906A1 (en) 2002-03-28
ES2628349T3 (es) 2017-08-02
DK2594640T3 (en) 2016-03-21
HK1188249A1 (en) 2014-04-25
EP2594641A1 (de) 2013-05-22
JP2014065733A (ja) 2014-04-17
CY1119026T1 (el) 2018-01-10
EP2594640B1 (de) 2015-12-30
CY1117521T1 (el) 2017-04-26
JP6425775B2 (ja) 2018-11-21
EP2801618A1 (de) 2014-11-12
ES2561293T3 (es) 2016-02-25
CY1118972T1 (el) 2018-01-10
ES2561292T3 (es) 2016-02-25
EP2636740A1 (de) 2013-09-11
EP2594642B1 (de) 2015-12-30
EP2602322A1 (de) 2013-06-12
ES2315788T5 (es) 2020-09-17
US20080209581A1 (en) 2008-08-28
EP2940139B1 (de) 2018-07-04
PT1619249E (pt) 2009-01-07
EP2940139A1 (de) 2015-11-04
AU2002211062C1 (en) 2011-02-03
PT2636741E (pt) 2016-06-09
AU2011201325B2 (en) 2011-12-22
HK1184820A1 (en) 2014-01-30
US20150361424A1 (en) 2015-12-17
US7973015B2 (en) 2011-07-05
EP3382021A1 (de) 2018-10-03
US20030235845A1 (en) 2003-12-25
PT2284264T (pt) 2017-03-06
CY1117522T1 (el) 2017-04-26
ES2609421T3 (es) 2017-04-20
DK2594642T3 (en) 2016-03-21
HK1184818A1 (en) 2014-01-30

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