JP7042255B2 - ヒトｌｍｎａを標的にするオリゴヌクレオチド類縁体 - Google Patents

ヒトｌｍｎａを標的にするオリゴヌクレオチド類縁体 Download PDF

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JP7042255B2
JP7042255B2 JP2019508165A JP2019508165A JP7042255B2 JP 7042255 B2 JP7042255 B2 JP 7042255B2 JP 2019508165 A JP2019508165 A JP 2019508165A JP 2019508165 A JP2019508165 A JP 2019508165A JP 7042255 B2 JP7042255 B2 JP 7042255B2
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oligomers
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sequence
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JP2019514426A (ja
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マイケル アール． エルドス，
フランシス エス． コリンズ，
カン カオ，
リシャルド コール，
リチャード キース ベストウィック，
レスリー ビー． ゴードン，
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United States Department of Health and Human Services
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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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    • C12N2310/30—Chemical structure
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    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C12N2310/3183—Diol linkers, e.g. glycols or propanediols
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Plant Pathology (AREA)
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  • Urology & Nephrology (AREA)
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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JP2019508165A 2016-04-29 2017-04-28 ヒトｌｍｎａを標的にするオリゴヌクレオチド類縁体 Active JP7042255B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2022000125A JP2022046725A (ja) 2016-04-29 2022-01-04 ヒトｌｍｎａを標的にするオリゴヌクレオチド類縁体

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662330027P 2016-04-29 2016-04-29
US62/330,027 2016-04-29
PCT/US2017/030174 WO2017190041A1 (en) 2016-04-29 2017-04-28 Oligonucleotide analogues targeting human lmna

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JP2019514426A JP2019514426A (ja) 2019-06-06
JP2019514426A5 JP2019514426A5 (enExample) 2020-05-21
JP7042255B2 true JP7042255B2 (ja) 2022-03-25

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JP2022000125A Pending JP2022046725A (ja) 2016-04-29 2022-01-04 ヒトｌｍｎａを標的にするオリゴヌクレオチド類縁体

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US (2) US10822608B2 (enExample)
EP (1) EP3449000A1 (enExample)
JP (2) JP7042255B2 (enExample)
KR (1) KR102329187B1 (enExample)
CN (1) CN109477109B (enExample)
AU (3) AU2017258642B2 (enExample)
CA (1) CA3022303A1 (enExample)
CO (1) CO2018012873A2 (enExample)
EA (1) EA201892467A1 (enExample)
MA (1) MA45616A (enExample)
MX (1) MX2018013157A (enExample)
NZ (1) NZ747685A (enExample)
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WO (1) WO2017190041A1 (enExample)

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