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WO2025117544A1 - Engineered omega guide molecule and iscb compositions, systems, and methods of use thereof - Google Patents

Engineered omega guide molecule and iscb compositions, systems, and methods of use thereof Download PDF

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Publication number
WO2025117544A1
WO2025117544A1 PCT/US2024/057461 US2024057461W WO2025117544A1 WO 2025117544 A1 WO2025117544 A1 WO 2025117544A1 US 2024057461 W US2024057461 W US 2024057461W WO 2025117544 A1 WO2025117544 A1 WO 2025117544A1
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swa
systems
compositions
com com
engineered
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French (fr)
Inventor
Feng Zhang
Han ALTAE-TRAN
Soumya KANNAN
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Massachusetts Institute of Technology
Broad Institute Inc
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Massachusetts Institute of Technology
Broad Institute Inc
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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/195Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from bacteria
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3519Fusion with another nucleic acid

Definitions

  • ene seuuee .. are arence aren -o- re re-o- -o- re re-o- nosarone eanree saeurueeence aren -o- re re-o- -o- re re-o- nosarone eanree saeurueeence aren -o- re re-o- -o- re re-o- nosarone eanree saeurueeence aren -o- re re-o- -o- re re-o- nosarone eanree saeurueeence aren -o- re re-o- -o- re re-o- nosarone eanree saeurueeence aren -o- re re-o- -o- nosarone eanree saeurueeence aren -o-

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Absorbent Articles And Supports Therefor (AREA)
  • Prostheses (AREA)
  • Materials For Medical Uses (AREA)

Abstract

The present disclosure provides engineered OMEGA guide molecules, trans-oligonucleotide activated OMEGA guide systems, and compositions and systems comprising engineered IscB polypeptides. Methods of modifying target polynucleotides using the compositions and systems described herein are also provided.

Description

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Claims

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. – – . . , . . , . . , . . , . . – .– . . , . , . . , . ,. . , , ., , , , .,
. . ,. , , ,, , ,, - , - , , , , , , , , , , , , , , , , , , , , , - , , , , , , , , ’, , , , , , , ,,’, , , , , , , , , - , , ,, , , , , , , , , , - , , , , , ,-,’ , , ,, , , , , , , , , , , , , , , , ,, ,, , , , , , ,, , , , , , ,- , , , , , , ,, , . ,,. . , ,
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