KR20240037299A - Crispr/cas 편집 시스템용 가이드 rna - Google Patents

Crispr/cas 편집 시스템용 가이드 rna Download PDF

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Publication number
KR20240037299A
KR20240037299A KR1020247005580A KR20247005580A KR20240037299A KR 20240037299 A KR20240037299 A KR 20240037299A KR 1020247005580 A KR1020247005580 A KR 1020247005580A KR 20247005580 A KR20247005580 A KR 20247005580A KR 20240037299 A KR20240037299 A KR 20240037299A
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South Korea
Prior art keywords
grna
adenosine deaminase
nls
composition
amino acid
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Pending
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KR1020247005580A
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English (en)
Korean (ko)
Inventor
브라이언 캐퍼티
마이클 패커
이본 아라틴-샤우스
로-아이 청
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빔 테라퓨틱스, 인크.
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Publication of KR20240037299A publication Critical patent/KR20240037299A/ko
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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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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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
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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
    • 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
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
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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]
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
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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
    • C12N2310/34—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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3513—Protein; Peptide
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/34—Allele or polymorphism specific uses

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  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Diabetes (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
KR1020247005580A 2021-07-23 2022-07-22 Crispr/cas 편집 시스템용 가이드 rna Pending KR20240037299A (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202163225322P 2021-07-23 2021-07-23
US63/225,322 2021-07-23
US202163255927P 2021-10-14 2021-10-14
US63/255,927 2021-10-14
PCT/US2022/074041 WO2023004409A1 (en) 2021-07-23 2022-07-22 Guide rnas for crispr/cas editing systems

Publications (1)

Publication Number Publication Date
KR20240037299A true KR20240037299A (ko) 2024-03-21

Family

ID=83149172

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020247005580A Pending KR20240037299A (ko) 2021-07-23 2022-07-22 Crispr/cas 편집 시스템용 가이드 rna

Country Status (7)

Country Link
US (1) US20240301405A1 (https=)
EP (1) EP4373931A1 (https=)
JP (1) JP2024529425A (https=)
KR (1) KR20240037299A (https=)
AU (1) AU2022313315A1 (https=)
CA (1) CA3226664A1 (https=)
WO (1) WO2023004409A1 (https=)

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US20230383277A1 (en) * 2020-10-14 2023-11-30 Beam Therapeutics Inc. Compositions and methods for treating glycogen storage disease type 1a
WO2024220980A2 (en) * 2023-04-20 2024-10-24 The Schepens Eye Research Institute, Inc. Dscam inhibitory nucleic acids and methods of use thereof
IL324634A (en) 2023-05-15 2026-01-01 Nchroma Bio Inc Compositions and methods for epigenetic regulation of hbv gene expression
WO2025087411A1 (zh) * 2023-10-25 2025-05-01 尧唐(上海)生物科技有限公司 一种脱氨酶、包含其的碱基编辑器及其应用

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US4880635B1 (en) 1984-08-08 1996-07-02 Liposome Company Dehydrated liposomes
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
US4911928A (en) 1987-03-13 1990-03-27 Micro-Pak, Inc. Paucilamellar lipid vesicles
US4917951A (en) 1987-07-28 1990-04-17 Micro-Pak, Inc. Lipid vesicles formed of surfactants and steroids
US5846946A (en) 1996-06-14 1998-12-08 Pasteur Merieux Serums Et Vaccins Compositions and methods for administering Borrelia DNA
JP4772045B2 (ja) 2004-07-16 2011-09-14 アメリカ合衆国 Ｃｍｖ／ｒ核酸コンストラクトを含むａｉｄｓに対するワクチン
WO2009088786A1 (en) 2007-12-31 2009-07-16 Nanocor Therapeutics, Inc. Rna interference for the treatment of heart failure
EP3241902B1 (en) 2012-05-25 2018-02-28 The Regents of The University of California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
CN105139759B (zh) 2015-09-18 2017-10-10 京东方科技集团股份有限公司 一种拼接屏
WO2017058751A1 (en) * 2015-09-28 2017-04-06 North Carolina State University Methods and compositions for sequence specific antimicrobials
JP7109784B2 (ja) 2015-10-23 2022-08-01 プレジデント アンド フェローズ オブ ハーバード カレッジ 遺伝子編集のための進化したＣａｓ９蛋白質
KR102547316B1 (ko) 2016-08-03 2023-06-23 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
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Publication number Publication date
AU2022313315A1 (en) 2024-02-08
US20240301405A1 (en) 2024-09-12
CA3226664A1 (en) 2023-01-26
WO2023004409A1 (en) 2023-01-26
JP2024529425A (ja) 2024-08-06
EP4373931A1 (en) 2024-05-29

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