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 (enExample
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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]
    • C—CHEMISTRY; METALLURGY
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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
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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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  • Microbiology (AREA)
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  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (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 (enExample) 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

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US (3) US11479767B2 (enExample)
EP (1) EP3551757A1 (enExample)
JP (3) 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) MX2024001138A (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)

Families Citing this family (134)

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