KR20150070278A - C9orf72 발현 조절용 조성물 - Google Patents

C9orf72 발현 조절용 조성물 Download PDF

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Publication number
KR20150070278A
KR20150070278A KR1020157012430A KR20157012430A KR20150070278A KR 20150070278 A KR20150070278 A KR 20150070278A KR 1020157012430 A KR1020157012430 A KR 1020157012430A KR 20157012430 A KR20157012430 A KR 20157012430A KR 20150070278 A KR20150070278 A KR 20150070278A
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Prior art keywords
compound
c9orf72
exon
certain embodiments
nucleic acid
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Abandoned
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KR1020157012430A
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Korean (ko)
Inventor
프랑크 씨. 벤네트
수잔 엠. 프레이어
에릭 이. 스와이제
프랑크 리고
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아이시스 파마수티컬즈 인코포레이티드
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Publication of KR20150070278A publication Critical patent/KR20150070278A/ko
Abandoned legal-status Critical Current

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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
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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • 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
    • 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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    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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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
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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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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
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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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  • Chemical & Material Sciences (AREA)
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  • General Health & Medical Sciences (AREA)
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  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
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  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychology (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)
  • Saccharide Compounds (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
KR1020157012430A 2012-10-15 2013-10-15 C9orf72 발현 조절용 조성물 Abandoned KR20150070278A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261714132P 2012-10-15 2012-10-15
US61/714,132 2012-10-15
PCT/US2013/065073 WO2014062691A2 (en) 2012-10-15 2013-10-15 Compositions for modulating c9orf72 expression

Publications (1)

Publication Number Publication Date
KR20150070278A true KR20150070278A (ko) 2015-06-24

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KR1020157012430A Abandoned KR20150070278A (ko) 2012-10-15 2013-10-15 C9orf72 발현 조절용 조성물

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Country Link
US (3) US10443052B2 (enExample)
EP (1) EP2906258A4 (enExample)
JP (3) JP6570447B2 (enExample)
KR (1) KR20150070278A (enExample)
CN (2) CN104968783B (enExample)
AU (2) AU2013331434B2 (enExample)
BR (1) BR112015008399A8 (enExample)
CA (1) CA2888486A1 (enExample)
HK (1) HK1212597A1 (enExample)
IL (1) IL238266A0 (enExample)
MX (2) MX370924B (enExample)
RU (2) RU2730677C2 (enExample)
WO (1) WO2014062691A2 (enExample)

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