JP7696694B2 - 修飾されたガイドｒｎａ - Google Patents

修飾されたガイドｒｎａ Download PDF

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JP7696694B2
JP7696694B2 JP2019530394A JP2019530394A JP7696694B2 JP 7696694 B2 JP7696694 B2 JP 7696694B2 JP 2019530394 A JP2019530394 A JP 2019530394A JP 2019530394 A JP2019530394 A JP 2019530394A JP 7696694 B2 JP7696694 B2 JP 7696694B2
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nucleotides
modified
sgrna
region
hairpin
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JP2019536464A5 (enExample
JP2019536464A (ja
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マディソン ローデン スミス，エイミー
ブイ． モリッシー，デイビッド
ストラップス，ウォルター
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インテリア セラピューティクス，インコーポレイテッド
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Priority to JP2024158151A priority patent/JP7899270B2/ja
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    • C—CHEMISTRY; METALLURGY
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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
    • 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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    • C12N15/00—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
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • 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
    • C12N2310/31—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
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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
    • 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
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
    • C—CHEMISTRY; METALLURGY
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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
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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
    • C12N2310/3525—MOE, methoxyethoxy
    • 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
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
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  • Chemical & Material Sciences (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
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  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Saccharide Compounds (AREA)
  • Cosmetics (AREA)
JP2019530394A 2016-12-08 2017-12-08 修飾されたガイドｒｎａ Active JP7696694B2 (ja)

Priority Applications (3)

Application Number Priority Date Filing Date Title
JP2022096277A JP7625555B2 (ja) 2016-12-08 2022-06-15 修飾されたガイドｒｎａ
JP2024158151A JP7899270B2 (ja) 2016-12-08 2024-09-12 修飾されたガイドｒｎａ
JP2026036119A JP2026097966A (ja) 2016-12-08 2026-03-06 修飾されたガイドｒｎａ

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662431756P 2016-12-08 2016-12-08
US62/431,756 2016-12-08
PCT/US2017/065306 WO2018107028A1 (en) 2016-12-08 2017-12-08 Modified guide rnas

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2022096277A Division JP7625555B2 (ja) 2016-12-08 2022-06-15 修飾されたガイドｒｎａ

Publications (3)

Publication Number Publication Date
JP2019536464A JP2019536464A (ja) 2019-12-19
JP2019536464A5 JP2019536464A5 (enExample) 2021-01-21
JP7696694B2 true JP7696694B2 (ja) 2025-06-23

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ID=60937874

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Application Number Title Priority Date Filing Date
JP2019530394A Active JP7696694B2 (ja) 2016-12-08 2017-12-08 修飾されたガイドｒｎａ
JP2022096277A Active JP7625555B2 (ja) 2016-12-08 2022-06-15 修飾されたガイドｒｎａ
JP2024158151A Active JP7899270B2 (ja) 2016-12-08 2024-09-12 修飾されたガイドｒｎａ
JP2026036119A Pending JP2026097966A (ja) 2016-12-08 2026-03-06 修飾されたガイドｒｎａ

Family Applications After (3)

Application Number Title Priority Date Filing Date
JP2022096277A Active JP7625555B2 (ja) 2016-12-08 2022-06-15 修飾されたガイドｒｎａ
JP2024158151A Active JP7899270B2 (ja) 2016-12-08 2024-09-12 修飾されたガイドｒｎａ
JP2026036119A Pending JP2026097966A (ja) 2016-12-08 2026-03-06 修飾されたガイドｒｎａ

Country Status (18)

Country Link
US (4) US11479767B2 (enExample)
EP (1) EP3551757A1 (enExample)
JP (4) JP7696694B2 (enExample)
KR (1) KR102595683B1 (enExample)
CN (2) CN119464289A (enExample)
AU (2) AU2017374044B2 (enExample)
BR (1) BR112019011509A2 (enExample)
CA (1) CA3046376A1 (enExample)
CO (1) CO2019007258A2 (enExample)
EA (1) EA201991369A1 (enExample)
IL (1) IL267024B2 (enExample)
MX (2) MX2025000703A (enExample)
MY (1) MY206324A (enExample)
PH (1) PH12019501262A1 (enExample)
SG (1) SG10202106058WA (enExample)
TW (1) TWI835719B (enExample)
WO (1) WO2018107028A1 (enExample)
ZA (1) ZA202007633B (enExample)

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