JP2016528897A - Ｒｎａを調節するための組成物および方法 - Google Patents

Ｒｎａを調節するための組成物および方法 Download PDF

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JP2016528897A
JP2016528897A JP2016534875A JP2016534875A JP2016528897A JP 2016528897 A JP2016528897 A JP 2016528897A JP 2016534875 A JP2016534875 A JP 2016534875A JP 2016534875 A JP2016534875 A JP 2016534875A JP 2016528897 A JP2016528897 A JP 2016528897A
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oligonucleotide
rna
nucleotides
complementary
nucleotide
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JP2016528897A5 (enExample
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ファティー オゾラック，
ファティー オゾラック，
キャロライン ウー，
キャロライン ウー，
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ラナ セラピューティクス インコーポレイテッド
ラナ セラピューティクス インコーポレイテッド
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    • C—CHEMISTRY; METALLURGY
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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
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    • C12N15/09—Recombinant DNA-technology
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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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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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
    • C12N15/67—General methods for enhancing the expression
    • 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
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
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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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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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  • Health & Medical Sciences (AREA)
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  • Engineering & Computer Science (AREA)
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  • Chemical & Material Sciences (AREA)
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  • Molecular Biology (AREA)
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  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
JP2016534875A 2013-08-16 2014-08-15 Ｒｎａを調節するための組成物および方法 Pending JP2016528897A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201361866989P 2013-08-16 2013-08-16
US61/866,989 2013-08-16
US201361898461P 2013-10-31 2013-10-31
US61/898,461 2013-10-31
US201462010417P 2014-06-10 2014-06-10
US62/010,417 2014-06-10
PCT/US2014/051331 WO2015023975A1 (en) 2013-08-16 2014-08-15 Compositions and methods for modulating rna

Publications (2)

Publication Number Publication Date
JP2016528897A true JP2016528897A (ja) 2016-09-23
JP2016528897A5 JP2016528897A5 (enExample) 2017-09-28

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US (8) US20150050738A1 (enExample)
EP (1) EP3033424A4 (enExample)
JP (1) JP2016528897A (enExample)
KR (1) KR20160036065A (enExample)
CN (1) CN105658797A (enExample)
AU (2) AU2014306416B2 (enExample)
BR (1) BR112016003127A2 (enExample)
CA (1) CA2921556A1 (enExample)
EA (1) EA201690403A1 (enExample)
IL (1) IL244081A0 (enExample)
MX (1) MX2016002044A (enExample)
SG (1) SG11201600987TA (enExample)
WO (1) WO2015023975A1 (enExample)

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