KR20240099164A - Pah-조절 조성물 및 방법 - Google Patents

Pah-조절 조성물 및 방법

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Publication number
KR20240099164A
KR20240099164A KR1020247011690A KR20247011690A KR20240099164A KR 20240099164 A KR20240099164 A KR 20240099164A KR 1020247011690 A KR1020247011690 A KR 1020247011690A KR 20247011690 A KR20247011690 A KR 20247011690A KR 20240099164 A KR20240099164 A KR 20240099164A
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KR
South Korea
Prior art keywords
sequence
domain
grna
template rna
genetic modification
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
KR1020247011690A
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English (en)
Korean (ko)
Inventor
로버트 찰스 알트슐러
앤 헬렌 보트머
다니엘 레이먼드 치
세실리아 지오반나 실비아 코타-라무시노
규식 김
랜디 미쉘 코틀라
그레고리 데이비드 맥앨리스터
아나냐 레이
나타니엘 로케트
카를로스 산체스
배럿 에단 스타인버그
윌리엄 에드워드 살로몬
로버트 제임스 시토리크
윌리엄 쿼르베스
루치아노 헨리케 아포니
잔 왕
Original Assignee
플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
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Publication date
Application filed by 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 filed Critical 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨
Publication of KR20240099164A publication Critical patent/KR20240099164A/ko
Pending legal-status Critical Current

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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
    • C12N15/1137—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 against enzymes
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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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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N9/0004—Oxidoreductases (1.)
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    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1241—Nucleotidyltransferases (2.7.7)
    • C12N9/1276—RNA-directed DNA polymerase (2.7.7.49), i.e. reverse transcriptase or telomerase
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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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    • C12Y114/00—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
    • C12Y114/16—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with reduced pteridine as one donor, and incorporation of one atom of oxygen (1.14.16)
    • C12Y114/16001—Phenylalanine 4-monooxygenase (1.14.16.1)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
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    • C12N2310/00—Structure or type of the nucleic acid
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  • Health & Medical Sciences (AREA)
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  • Pharmacology & Pharmacy (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Virology (AREA)
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  • Cell Biology (AREA)
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  • Crystallography & Structural Chemistry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
KR1020247011690A 2021-09-08 2022-09-07 Pah-조절 조성물 및 방법 Pending KR20240099164A (ko)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202163241897P 2021-09-08 2021-09-08
US63/241,897 2021-09-08
US202263303927P 2022-01-27 2022-01-27
US63/303,927 2022-01-27
US202263367025P 2022-06-24 2022-06-24
US63/367,025 2022-06-24
PCT/US2022/076058 WO2023039435A2 (en) 2021-09-08 2022-09-07 Pah-modulating compositions and methods

Publications (1)

Publication Number Publication Date
KR20240099164A true KR20240099164A (ko) 2024-06-28

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KR1020247011690A Pending KR20240099164A (ko) 2021-09-08 2022-09-07 Pah-조절 조성물 및 방법

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Country Link
US (1) US12544458B2 (https=)
EP (1) EP4399306A4 (https=)
JP (1) JP2024533315A (https=)
KR (1) KR20240099164A (https=)
AU (1) AU2022342168A1 (https=)
CA (1) CA3231712A1 (https=)
IL (1) IL311218A (https=)
TW (1) TW202320810A (https=)
WO (1) WO2023039435A2 (https=)

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