TW202320864A - 經工程改造之casx抑制子系統 - Google Patents

經工程改造之casx抑制子系統

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Publication number
TW202320864A
TW202320864A TW111135799A TW111135799A TW202320864A TW 202320864 A TW202320864 A TW 202320864A TW 111135799 A TW111135799 A TW 111135799A TW 111135799 A TW111135799 A TW 111135799A TW 202320864 A TW202320864 A TW 202320864A
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Taiwan
Prior art keywords
seq
sequence
gene
domain
grna
Prior art date
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TW111135799A
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English (en)
Chinese (zh)
Inventor
傑森 費爾南德斯
羅斯 懷特
尚恩 希金斯
莎拉 丹妮
班傑明 奧克斯
埃梅里克 珍 馬里烏斯 查爾斯
Original Assignee
美商斯奎柏治療公司
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Publication of TW202320864A publication Critical patent/TW202320864A/zh

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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]
    • 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
    • A61K48/005—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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    • C12N15/11—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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    • 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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    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88—Introduction 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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    • C12N15/87—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
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    • C12N9/10—Transferases (2.)
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    • C12N9/1007—Methyltransferases (general) (2.1.1.)
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    • C07K2319/00—Fusion polypeptide
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    • C07K2319/00—Fusion polypeptide
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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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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Virology (AREA)
  • Pharmacology & Pharmacy (AREA)
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  • Cell Biology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Peptides Or Proteins (AREA)
  • Apparatus Associated With Microorganisms And Enzymes (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
TW111135799A 2021-09-21 2022-09-21 經工程改造之casx抑制子系統 TW202320864A (zh)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US202163246543P 2021-09-21 2021-09-21
US63/246,543 2021-09-21
US202263321517P 2022-03-18 2022-03-18
US63/321,517 2022-03-18

Publications (1)

Publication Number Publication Date
TW202320864A true TW202320864A (zh) 2023-06-01

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US (3) US20240392271A1 (enExample)
EP (1) EP4405479A2 (enExample)
JP (1) JP2024534523A (enExample)
KR (1) KR20240095525A (enExample)
AU (1) AU2022349627A1 (enExample)
CA (1) CA3231909A1 (enExample)
CL (1) CL2024000835A1 (enExample)
GB (1) GB2625500B (enExample)
IL (1) IL311610A (enExample)
MX (1) MX2024003455A (enExample)
PE (1) PE20240728A1 (enExample)
TW (1) TW202320864A (enExample)
WO (1) WO2023049742A2 (enExample)

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