TW202325848A - 用於基因體編輯之多核苷酸、組合物及方法 - Google Patents

用於基因體編輯之多核苷酸、組合物及方法 Download PDF

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Publication number
TW202325848A
TW202325848A TW111141875A TW111141875A TW202325848A TW 202325848 A TW202325848 A TW 202325848A TW 111141875 A TW111141875 A TW 111141875A TW 111141875 A TW111141875 A TW 111141875A TW 202325848 A TW202325848 A TW 202325848A
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Taiwan
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polynucleotide
nucleotides
sequence
orf
seq
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TW111141875A
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English (en)
Chinese (zh)
Inventor
薩賓 莫雷帕帝
琳得喜 珍 史崔特茲
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美商英特利亞醫療公司
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Publication of TW202325848A publication Critical patent/TW202325848A/zh

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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]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008Medicinal 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
    • A61K48/0016Medicinal 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 wherein the nucleic acid is delivered as a 'naked' nucleic acid, i.e. not combined with an entity such as a cationic lipid
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K9/50Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
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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
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
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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
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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
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
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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
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • C12N15/902Stable introduction of foreign DNA into chromosome using homologous recombination
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    • C12N9/14Hydrolases (3)
    • C12N9/78Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
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    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/01Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
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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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    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/50Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
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    • C12YENZYMES
    • C12Y305/00Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04005Cytidine deaminase (3.5.4.5)

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  • Health & Medical Sciences (AREA)
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  • Genetics & Genomics (AREA)
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  • Biomedical Technology (AREA)
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  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
TW111141875A 2021-11-03 2022-11-02 用於基因體編輯之多核苷酸、組合物及方法 TW202325848A (zh)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US202163275425P 2021-11-03 2021-11-03
US63/275,425 2021-11-03
US202263352158P 2022-06-14 2022-06-14
US63/352,158 2022-06-14

Publications (1)

Publication Number Publication Date
TW202325848A true TW202325848A (zh) 2023-07-01

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US (1) US20240301377A1 (enExample)
EP (1) EP4426822A2 (enExample)
JP (1) JP2024542995A (enExample)
KR (1) KR20240114296A (enExample)
AU (1) AU2022382975A1 (enExample)
CA (1) CA3237303A1 (enExample)
CL (1) CL2024001332A1 (enExample)
CO (1) CO2024007019A2 (enExample)
IL (1) IL312508A (enExample)
MX (1) MX2024005242A (enExample)
TW (1) TW202325848A (enExample)
WO (1) WO2023081689A2 (enExample)

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WO2025137301A1 (en) * 2023-12-20 2025-06-26 Intellia Therapeutics, Inc. Methods for rapid engineering of cells

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JPH10500310A (ja) 1994-05-19 1998-01-13 ダコ アクティーゼルスカブ 淋菌及びトラコーマ クラミジアの検出のためのpna プローブ
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Publication number Publication date
EP4426822A2 (en) 2024-09-11
KR20240114296A (ko) 2024-07-23
US20240301377A1 (en) 2024-09-12
JP2024542995A (ja) 2024-11-19
IL312508A (en) 2024-07-01
CO2024007019A2 (es) 2024-06-07
CL2024001332A1 (es) 2024-11-15
AU2022382975A1 (en) 2024-05-02
CA3237303A1 (en) 2023-05-11
WO2023081689A2 (en) 2023-05-11
WO2023081689A3 (en) 2023-06-08
MX2024005242A (es) 2024-07-02

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