CN118345072A - 合成的指导分子、组合物和与其相关的方法 - Google Patents

合成的指导分子、组合物和与其相关的方法 Download PDF

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Publication number
CN118345072A
CN118345072A CN202410342599.0A CN202410342599A CN118345072A CN 118345072 A CN118345072 A CN 118345072A CN 202410342599 A CN202410342599 A CN 202410342599A CN 118345072 A CN118345072 A CN 118345072A
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guide molecule
guide
molecule
independently
integer
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Chinese (zh)
Inventor
J·海尔
S·金
S·萨科曼诺
S·凯普哈特
C·费尔南德斯
H·嘉亚拉穆
B·伊顿
K·Z·贝里
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Editas Medicine Inc
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Editas Medicine Inc
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/712Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Hydrogenated Pyridines (AREA)
  • Saccharide Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CN202410342599.0A 2016-12-30 2017-12-29 合成的指导分子、组合物和与其相关的方法 Pending CN118345072A (zh)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US201662441046P 2016-12-30 2016-12-30
US62/441,046 2016-12-30
US201762492001P 2017-04-28 2017-04-28
US62/492,001 2017-04-28
CN201780085248.4A CN110249052B (zh) 2016-12-30 2017-12-29 合成的指导分子、组合物和与其相关的方法
PCT/US2017/069019 WO2018126176A1 (en) 2016-12-30 2017-12-29 Synthetic guide molecules, compositions and methods relating thereto

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Publication Number Publication Date
CN118345072A true CN118345072A (zh) 2024-07-16

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CN201780085248.4A Active CN110249052B (zh) 2016-12-30 2017-12-29 合成的指导分子、组合物和与其相关的方法

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US (1) US20230111575A1 (https=)
EP (1) EP3565895A1 (https=)
JP (3) JP7167029B2 (https=)
KR (2) KR102758180B1 (https=)
CN (2) CN118345072A (https=)
AU (2) AU2017388753A1 (https=)
CA (1) CA3048434A1 (https=)
MX (1) MX420023B (https=)
WO (1) WO2018126176A1 (https=)

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JP2025016776A (ja) 2025-02-04
AU2024205204A1 (en) 2024-09-05
MX420023B (es) 2025-01-14
JP2022173349A (ja) 2022-11-18
KR102758180B1 (ko) 2025-01-23
JP2020503049A (ja) 2020-01-30
US20230111575A1 (en) 2023-04-13
WO2018126176A1 (en) 2018-07-05
JP7610559B2 (ja) 2025-01-08
MX2019007750A (es) 2019-10-15
EP3565895A1 (en) 2019-11-13
KR20230175330A (ko) 2023-12-29
CN110249052B (zh) 2024-04-12
KR102618864B1 (ko) 2024-01-02
AU2017388753A1 (en) 2019-07-11
KR20190110554A (ko) 2019-09-30
JP7167029B2 (ja) 2022-11-08
CN110249052A (zh) 2019-09-17
CA3048434A1 (en) 2018-07-05

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