AU2022349627A1 - Engineered casx repressor systems - Google Patents

Engineered casx repressor systems Download PDF

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Publication number
AU2022349627A1
AU2022349627A1 AU2022349627A AU2022349627A AU2022349627A1 AU 2022349627 A1 AU2022349627 A1 AU 2022349627A1 AU 2022349627 A AU2022349627 A AU 2022349627A AU 2022349627 A AU2022349627 A AU 2022349627A AU 2022349627 A1 AU2022349627 A1 AU 2022349627A1
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Australia
Prior art keywords
seq
sequence
gene
domain
repressor system
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AU2022349627A
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Inventor
Emeric Jean Marius CHARLES
Sarah DENNY
Jason Fernandes
Sean Higgins
Benjamin OAKES
Ross White
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Scribe Therapeutics Inc
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Scribe Therapeutics Inc
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    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • A—HUMAN NECESSITIES
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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
    • C12N15/11—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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    • C12N15/63—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
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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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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    • C12N15/09—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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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • 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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    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
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    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
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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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  • Animal Behavior & Ethology (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)
AU2022349627A 2021-09-21 2022-09-21 Engineered casx repressor systems Pending AU2022349627A1 (en)

Applications Claiming Priority (5)

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
PCT/US2022/076774 WO2023049742A2 (en) 2021-09-21 2022-09-21 Engineered casx repressor systems

Publications (1)

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AU2022349627A1 true AU2022349627A1 (en) 2024-03-21

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AU2022349627A Pending AU2022349627A1 (en) 2021-09-21 2022-09-21 Engineered casx repressor systems

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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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