HRP20151053T1 - Metoda za uäśinkovito preskakanje eksona (44) u duchenne mišiä†noj distrofiji i srodnim namjenama - Google Patents

Metoda za uäśinkovito preskakanje eksona (44) u duchenne mišiä†noj distrofiji i srodnim namjenama Download PDF

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Publication number
HRP20151053T1
HRP20151053T1 HRP20151053TT HRP20151053T HRP20151053T1 HR P20151053 T1 HRP20151053 T1 HR P20151053T1 HR P20151053T T HRP20151053T T HR P20151053TT HR P20151053 T HRP20151053 T HR P20151053T HR P20151053 T1 HRP20151053 T1 HR P20151053T1
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Croatia
Prior art keywords
oligonucleotide
vector
pharmaceutical composition
skipping
muscular dystrophy
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HRP20151053TT
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English (en)
Croatian (hr)
Inventor
Gerardus Johannes Platenburg
Josephus Johannes De Kimpe
Judith Christina Theodora Van Deutekom
Garrit-Jan Boudewijn Van Ommen
Annemieke Aartsma-Rus
Original Assignee
Prosensa Technologies B.V.
Academisch Ziekenhuis Leiden
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Application filed by Prosensa Technologies B.V., Academisch Ziekenhuis Leiden filed Critical Prosensa Technologies B.V.
Publication of HRP20151053T1 publication Critical patent/HRP20151053T1/hr

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    • 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
    • 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
    • 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
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/31—Chemical structure of the backbone
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3233—Morpholino-type ring
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/3513—Protein; Peptide
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/3517—Marker; Tag
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    • 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
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Wood Science & Technology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Plant Pathology (AREA)
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  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Ultra Sonic Daignosis Equipment (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
HRP20151053TT 2008-05-14 2009-05-14 Metoda za uäśinkovito preskakanje eksona (44) u duchenne mišiä†noj distrofiji i srodnim namjenama HRP20151053T1 (hr)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
EP08156193A EP2119783A1 (en) 2008-05-14 2008-05-14 Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means
US12801008P 2008-05-15 2008-05-15
PCT/NL2009/050258 WO2009139630A2 (en) 2008-05-14 2009-05-14 Method for efficient exon (44) skipping in duchenne muscular dystrophy and associated means
EP09746807.8A EP2283134B1 (en) 2008-05-14 2009-05-14 Method for efficient exon (44) skipping in duchenne muscular dystrophy and associated means

Publications (1)

Publication Number Publication Date
HRP20151053T1 true HRP20151053T1 (hr) 2016-01-29

Family

ID=39800733

Family Applications (2)

Application Number Title Priority Date Filing Date
HRP20151053TT HRP20151053T1 (hr) 2008-05-14 2009-05-14 Metoda za uäśinkovito preskakanje eksona (44) u duchenne mišiä†noj distrofiji i srodnim namjenama
HRP20171980TT HRP20171980T1 (hr) 2008-05-14 2017-12-20 Postupak djelotvornog preskakanja egzona (44) kod duchenneove mišićne distrofije i odgovarajuće sredstvo

Family Applications After (1)

Application Number Title Priority Date Filing Date
HRP20171980TT HRP20171980T1 (hr) 2008-05-14 2017-12-20 Postupak djelotvornog preskakanja egzona (44) kod duchenneove mišićne distrofije i odgovarajuće sredstvo

Country Status (17)

Country Link
US (4) US9139828B2 (enExample)
EP (5) EP2119783A1 (enExample)
JP (2) JP5696039B2 (enExample)
CN (1) CN102171342B (enExample)
AU (5) AU2009247037B2 (enExample)
CA (3) CA3048624C (enExample)
CY (1) CY1117098T1 (enExample)
DK (2) DK2813570T3 (enExample)
ES (3) ES2551154T3 (enExample)
HR (2) HRP20151053T1 (enExample)
HU (2) HUE038111T2 (enExample)
IL (6) IL316692A (enExample)
LT (1) LT2813570T (enExample)
NZ (2) NZ589230A (enExample)
PL (2) PL2283134T3 (enExample)
PT (2) PT2283134E (enExample)
WO (1) WO2009139630A2 (enExample)

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