JP7043169B2 - イントロン保持の減少 - Google Patents

イントロン保持の減少 Download PDF

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JP7043169B2
JP7043169B2 JP2016573805A JP2016573805A JP7043169B2 JP 7043169 B2 JP7043169 B2 JP 7043169B2 JP 2016573805 A JP2016573805 A JP 2016573805A JP 2016573805 A JP2016573805 A JP 2016573805A JP 7043169 B2 JP7043169 B2 JP 7043169B2
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seq
polynucleic acid
acid polymer
intron
sequence
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JP2017519766A (ja
JP2017519766A5 (enExample
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ヴォアチョフスキー，イゴール
クラロヴィコヴァ，ヤナ
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ユニバーシティ・オブ・サザンプトン
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    • 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
    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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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
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • 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
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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
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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
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    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
    • C—CHEMISTRY; METALLURGY
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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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C12N2310/113—Antisense targeting other non-coding nucleic acids, e.g. antagomirs
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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
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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
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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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  • Diabetes (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
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  • Hematology (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2016573805A 2014-06-16 2015-06-16 イントロン保持の減少 Active JP7043169B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2020131686A JP7532136B2 (ja) 2014-06-16 2020-08-03 イントロン保持の減少

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GBGB1410693.4A GB201410693D0 (en) 2014-06-16 2014-06-16 Splicing modulation
GB1410693.4 2014-06-16
PCT/GB2015/051756 WO2015193651A1 (en) 2014-06-16 2015-06-16 Reducing intron retention

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JP2017519766A JP2017519766A (ja) 2017-07-20
JP2017519766A5 JP2017519766A5 (enExample) 2018-08-02
JP7043169B2 true JP7043169B2 (ja) 2022-03-29

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JP2020131686A Active JP7532136B2 (ja) 2014-06-16 2020-08-03 イントロン保持の減少
JP2022178219A Pending JP2023011885A (ja) 2014-06-16 2022-11-07 イントロン保持の減少

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JP2022178219A Pending JP2023011885A (ja) 2014-06-16 2022-11-07 イントロン保持の減少

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US (6) US9745577B2 (enExample)
EP (2) EP4015648B1 (enExample)
JP (3) JP7043169B2 (enExample)
KR (1) KR102638276B1 (enExample)
CN (1) CN107073137A (enExample)
AU (2) AU2015275870B2 (enExample)
CA (1) CA2951208A1 (enExample)
ES (1) ES2907033T3 (enExample)
GB (1) GB201410693D0 (enExample)
IL (2) IL295051B2 (enExample)
SG (2) SG11201610480XA (enExample)
WO (1) WO2015193651A1 (enExample)

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Ｈｕｍ Ｇｅｎｅｔ， ２０１０， Ｖｏｌ．１２８， ｐ．３８３－４００
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Ｍｏｌｅｃｕｌａｒ Ｔｈｅｒａｐｙ， ２００６， Ｖｏｌ．１４， Ｎｏ．４， ｐｐ．４７１－４７５
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ＳＵＥ ＦＬＥＴＣＨＥＲ，ＡＮＴＩＳＥＮＳＥ ＳＵＰＰＲＥＳＳＩＯＮ ＯＦ ＤＯＮＯＲ ＳＰＬＩＣＥ ＳＩＴＥ ＭＵＴＡＴＩＯＮＳ ＩＮ ＴＨＥ ＤＹＳＴＲＯＰＨＩＮ ＧＥＮＥ ＴＲＡＮＳＣＲＩＰＴ，ＭＯＬＥＣＵＬＡＲ ＧＥＮＥＴＩＣＳ ＆ ＧＥＮＯＭＩＣ ＭＥＤＩＣＩＮＥ，２０１３年 ６月１３日，ＶＯＬ：１， ＮＲ：３，ＰＡＧＥ（Ｓ）：１６２ － １７３，ＵＲＬ，ｈｔｔｐ：／／ｄｘ．ｄｏｉ．ｏｒｇ／１０．１００２／ｍｇｇ３．１９

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