DK2821085T3 - Rna-interferens til behandling af "gain-of-function"-forstyrrelser - Google Patents

Rna-interferens til behandling af "gain-of-function"-forstyrrelser Download PDF

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Publication number
DK2821085T3
DK2821085T3 DK14168602.2T DK14168602T DK2821085T3 DK 2821085 T3 DK2821085 T3 DK 2821085T3 DK 14168602 T DK14168602 T DK 14168602T DK 2821085 T3 DK2821085 T3 DK 2821085T3
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gain
treat
rna interference
function disorders
disorders
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DK14168602.2T
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Neil Aronin
Phillip D Zamore
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Univ Massachusetts
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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
    • C07H21/04—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 deoxyribosyl 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
    • C12N15/113—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
    • 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
    • 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
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2510/00—Genetically modified cells

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  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
DK14168602.2T 2003-09-12 2004-09-13 Rna-interferens til behandling af "gain-of-function"-forstyrrelser DK2821085T3 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US50267803P 2003-09-12 2003-09-12
EP04783980.8A EP1670518B1 (en) 2003-09-12 2004-09-13 Rna interference for the treatment of gain-of-function disorders

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Publication Number Publication Date
DK2821085T3 true DK2821085T3 (da) 2020-08-03

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US (1) US7947658B2 (da)
EP (3) EP1670518B1 (da)
CA (1) CA2579638C (da)
DK (1) DK2821085T3 (da)
ES (3) ES2969371T3 (da)
PT (1) PT2821085T (da)
WO (1) WO2005027980A1 (da)

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EP3760234A3 (en) 2021-04-07
WO2005027980A1 (en) 2005-03-31
EP1670518A4 (en) 2007-10-10
EP1670518B1 (en) 2014-05-21
EP3760234A2 (en) 2021-01-06
ES2808561T3 (es) 2021-03-01
ES2969371T3 (es) 2024-05-17
EP1670518A1 (en) 2006-06-21
EP2821085A2 (en) 2015-01-07
ES2485848T3 (es) 2014-08-14
PT2821085T (pt) 2020-07-28
EP2821085B1 (en) 2020-04-29
US20090118206A1 (en) 2009-05-07
US7947658B2 (en) 2011-05-24
EP3760234B1 (en) 2023-11-01
CA2579638A1 (en) 2005-03-31
EP2821085A3 (en) 2015-04-22
CA2579638C (en) 2016-04-19

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