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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JP2019536464A (ja
JP2019536464A5 (https=
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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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    • 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]
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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]
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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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
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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
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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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    • 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
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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
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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
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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
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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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  • Biotechnology (AREA)
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  • Microbiology (AREA)
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  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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)
  • Saccharide Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Cosmetics (AREA)
JP2019530394A 2016-12-08 2017-12-08 修飾されたガイドｒｎａ Active JP7696694B2 (ja)

Priority Applications (2)

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 JP2024178237A (ja) 2016-12-08 2024-09-12 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)

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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 (https=) 2021-01-21
JP7696694B2 true JP7696694B2 (ja) 2025-06-23

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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 Pending JP2024178237A (ja) 2016-12-08 2024-09-12 修飾されたガイドｒｎａ

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JP2022096277A Active JP7625555B2 (ja) 2016-12-08 2022-06-15 修飾されたガイドｒｎａ
JP2024158151A Pending JP2024178237A (ja) 2016-12-08 2024-09-12 修飾されたガイドｒｎａ

Country Status (18)

Country Link
US (4) US11479767B2 (https=)
EP (1) EP3551757A1 (https=)
JP (3) JP7696694B2 (https=)
KR (1) KR102595683B1 (https=)
CN (2) CN119464289A (https=)
AU (2) AU2017374044B2 (https=)
BR (1) BR112019011509A2 (https=)
CA (1) CA3046376A1 (https=)
CO (1) CO2019007258A2 (https=)
EA (1) EA201991369A1 (https=)
IL (1) IL267024B2 (https=)
MX (2) MX2024001138A (https=)
MY (1) MY206324A (https=)
PH (1) PH12019501262A1 (https=)
SG (1) SG10202106058WA (https=)
TW (1) TWI835719B (https=)
WO (1) WO2018107028A1 (https=)
ZA (1) ZA202007633B (https=)

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