KR20220123398A - 합성 가이드 rna, 이의 조성물, 방법 및 용도 - Google Patents

합성 가이드 rna, 이의 조성물, 방법 및 용도 Download PDF

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KR20220123398A
KR20220123398A KR1020227021479A KR20227021479A KR20220123398A KR 20220123398 A KR20220123398 A KR 20220123398A KR 1020227021479 A KR1020227021479 A KR 1020227021479A KR 20227021479 A KR20227021479 A KR 20227021479A KR 20220123398 A KR20220123398 A KR 20220123398A
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rna
nucleotides
stem
ligation
grna
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KR1020227021479A
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Korean (ko)
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브라이언 캐퍼티
아론 라슨
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빔 테라퓨틱스, 인크.
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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    • 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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    • 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
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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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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/93—Ligases (6)
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    • C12P—FERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
    • C12P19/00—Preparation of compounds containing saccharide radicals
    • C12P19/26—Preparation of nitrogen-containing carbohydrates
    • C12P19/28—N-glycosides
    • C12P19/30—Nucleotides
    • C12P19/34—Polynucleotides, e.g. nucleic acids, oligoribonucleotides
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    • C12Y—ENZYMES
    • C12Y605/00—Ligases forming phosphoric ester bonds (6.5)
    • C12Y605/01—Ligases forming phosphoric ester bonds (6.5) forming phosphoric ester bonds (6.5.1)
    • C12Y605/01003—RNA ligase (ATP) (6.5.1.3)
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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
    • 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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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
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  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
KR1020227021479A 2019-12-03 2020-12-03 합성 가이드 rna, 이의 조성물, 방법 및 용도 Pending KR20220123398A (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962943158P 2019-12-03 2019-12-03
US62/943,158 2019-12-03
US202063031262P 2020-05-28 2020-05-28
US63/031,262 2020-05-28
PCT/US2020/063084 WO2021113494A1 (en) 2019-12-03 2020-12-03 Synthetic guide rna, compositions, methods, and uses thereof

Publications (1)

Publication Number Publication Date
KR20220123398A true KR20220123398A (ko) 2022-09-06

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KR1020227021479A Pending KR20220123398A (ko) 2019-12-03 2020-12-03 합성 가이드 rna, 이의 조성물, 방법 및 용도

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Country Link
US (1) US20230055682A1 (https=)
EP (1) EP4069838A1 (https=)
JP (1) JP7826199B2 (https=)
KR (1) KR20220123398A (https=)
CN (2) CN121759549A (https=)
AU (1) AU2020398213A1 (https=)
CA (1) CA3162908A1 (https=)
WO (1) WO2021113494A1 (https=)

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CA3189601A1 (en) 2020-07-24 2022-01-27 The General Hospital Corporation Enhanced virus-like particles and methods of use thereof for delivery to cells
US20230220379A1 (en) * 2021-09-24 2023-07-13 Crispr Therapeutics Ag HIGH PURITY gRNA SYNTHESIS PROCESS
EP4441074A2 (en) 2021-12-03 2024-10-09 The Broad Institute, Inc. Compositions and methods for efficient in vivo delivery
EP4514953A4 (en) * 2022-04-25 2026-04-22 Huidagene Therapeutics Singapore Pte Ltd NEW CRISPR-CAS12F SYSTEMS AND THEIR USES
CN120303407A (zh) 2022-05-17 2025-07-11 恩维洛普治疗有限责任公司 用于有效体内递送的组合物和方法
WO2024003810A1 (en) * 2022-06-30 2024-01-04 Geneditbio Limited Guide rna with chemical modifications
WO2024102434A1 (en) 2022-11-10 2024-05-16 Senda Biosciences, Inc. Rna compositions comprising lipid nanoparticles or lipid reconstructed natural messenger packs
CN121079412A (zh) * 2023-04-28 2025-12-05 比姆医疗股份有限公司 经修饰的指导rna
CN117106782B (zh) * 2023-10-16 2024-04-26 吉林凯莱英医药化学有限公司 gRNA及其生物合成方法
WO2025114441A1 (en) * 2023-11-30 2025-06-05 F. Hoffmann-La Roche Ag Single guide rna synthesis
WO2025215514A1 (en) * 2024-04-08 2025-10-16 Crispr Therapeutics Ag Splint-mediated rna synthesis

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US4921757A (en) 1985-04-26 1990-05-01 Massachusetts Institute Of Technology System for delayed and pulsed release of biologically active substances
US4920016A (en) 1986-12-24 1990-04-24 Linear Technology, Inc. Liposomes with enhanced circulation time
JPH0825869B2 (ja) 1987-02-09 1996-03-13 株式会社ビタミン研究所 抗腫瘍剤包埋リポソ−ム製剤
US4917951A (en) 1987-07-28 1990-04-17 Micro-Pak, Inc. Lipid vesicles formed of surfactants and steroids
US4911928A (en) 1987-03-13 1990-03-27 Micro-Pak, Inc. Paucilamellar lipid vesicles
US5846946A (en) 1996-06-14 1998-12-08 Pasteur Merieux Serums Et Vaccins Compositions and methods for administering Borrelia DNA
AU2005274948B2 (en) 2004-07-16 2011-09-22 Genvec, Inc. Vaccines against aids comprising CMV/R-nucleic acid constructs
US8404658B2 (en) 2007-12-31 2013-03-26 Nanocor Therapeutics, Inc. RNA interference for the treatment of heart failure
US9187746B2 (en) * 2009-09-22 2015-11-17 Alnylam Pharmaceuticals, Inc. Dual targeting siRNA agents
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WO2021113264A1 (en) * 2019-12-02 2021-06-10 The Regents Of The University Of California Engineering circular guide rnas

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Publication number Publication date
JP2023504264A (ja) 2023-02-02
EP4069838A1 (en) 2022-10-12
JP7826199B2 (ja) 2026-03-09
AU2020398213A1 (en) 2022-06-16
CN121759549A (zh) 2026-03-31
US20230055682A1 (en) 2023-02-23
WO2021113494A1 (en) 2021-06-10
CA3162908A1 (en) 2021-06-10
CN114981426A (zh) 2022-08-30

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