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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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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
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    • 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
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (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)
  • Saccharide Compounds (AREA)
  • Hydrogenated Pyridines (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
PCT/US2017/069019 WO2018126176A1 (en) 2016-12-30 2017-12-29 Synthetic guide molecules, compositions and methods relating thereto
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

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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 (enExample)
EP (1) EP3565895A1 (enExample)
JP (3) JP7167029B2 (enExample)
KR (2) KR102618864B1 (enExample)
CN (2) CN118345072A (enExample)
AU (2) AU2017388753A1 (enExample)
CA (1) CA3048434A1 (enExample)
MX (1) MX420023B (enExample)
WO (1) WO2018126176A1 (enExample)

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

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