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WO2025171380A3 - Precision rna cleavage and editing using trans-cleaving ribozyme and guide rnas - Google Patents

Precision rna cleavage and editing using trans-cleaving ribozyme and guide rnas

Info

Publication number
WO2025171380A3
WO2025171380A3 PCT/US2025/015234 US2025015234W WO2025171380A3 WO 2025171380 A3 WO2025171380 A3 WO 2025171380A3 US 2025015234 W US2025015234 W US 2025015234W WO 2025171380 A3 WO2025171380 A3 WO 2025171380A3
Authority
WO
WIPO (PCT)
Prior art keywords
editing
trans
guide rnas
rna cleavage
cleaving ribozyme
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
PCT/US2025/015234
Other languages
French (fr)
Other versions
WO2025171380A2 (en
Inventor
Douglas Matthew ANDERSON
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
University of Rochester
Original Assignee
University of Rochester
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.)
Filing date
Publication date
Application filed by University of Rochester filed Critical University of Rochester
Publication of WO2025171380A2 publication Critical patent/WO2025171380A2/en
Publication of WO2025171380A3 publication Critical patent/WO2025171380A3/en
Pending legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/121—Hammerhead
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/123—Hepatitis delta
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another nucleic acid

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

Provided are compositions for site-specific cleavage, splicing and ligation of nucleic acid molecules including RNA and DNA, and methods of use of the compositions for generating spliced or ligated nucleic acid molecules and for the treatment of diseases.
PCT/US2025/015234 2024-02-09 2025-02-10 Precision rna cleavage and editing using trans-cleaving ribozyme and guide rnas Pending WO2025171380A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202463551572P 2024-02-09 2024-02-09
US63/551,572 2024-02-09

Publications (2)

Publication Number Publication Date
WO2025171380A2 WO2025171380A2 (en) 2025-08-14
WO2025171380A3 true WO2025171380A3 (en) 2025-09-18

Family

ID=94924641

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2025/015234 Pending WO2025171380A2 (en) 2024-02-09 2025-02-10 Precision rna cleavage and editing using trans-cleaving ribozyme and guide rnas

Country Status (1)

Country Link
WO (1) WO2025171380A2 (en)

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020210751A1 (en) * 2019-04-12 2020-10-15 The Broad Institute, Inc. System for genome editing
WO2021158964A1 (en) * 2020-02-07 2021-08-12 University Of Rochester Ribozyme-mediated rna assembly and expression
WO2023064895A1 (en) * 2021-10-15 2023-04-20 The Broad Institute, Inc. Rna-guided trans-splicing of rna

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2874751B2 (en) 1986-04-09 1999-03-24 ジェンザイム・コーポレーション Transgenic animals secreting the desired protein into milk
US4873316A (en) 1987-06-23 1989-10-10 Biogen, Inc. Isolation of exogenous recombinant proteins from the milk of transgenic mammals
US5703055A (en) 1989-03-21 1997-12-30 Wisconsin Alumni Research Foundation Generation of antibodies through lipid mediated DNA delivery
US5399346A (en) 1989-06-14 1995-03-21 The United States Of America As Represented By The Department Of Health And Human Services Gene therapy
US5585362A (en) 1989-08-22 1996-12-17 The Regents Of The University Of Michigan Adenovirus vectors for gene therapy
US5350674A (en) 1992-09-04 1994-09-27 Becton, Dickinson And Company Intrinsic factor - horse peroxidase conjugates and a method for increasing the stability thereof
HK1047109A1 (en) 1999-10-15 2003-02-07 University Of Massachusetts Rna interference pathway genes as tools for targeted genetic interference
US6326193B1 (en) 1999-11-05 2001-12-04 Cambria Biosciences, Llc Insect control agent
AU2001275474A1 (en) 2000-06-12 2001-12-24 Akkadix Corporation Materials and methods for the control of nematodes
EP2575895A2 (en) 2010-05-24 2013-04-10 Merck Sharp & Dohme Corp. Novel amino alcohol cationic lipids for oligonucleotide delivery

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020210751A1 (en) * 2019-04-12 2020-10-15 The Broad Institute, Inc. System for genome editing
WO2021158964A1 (en) * 2020-02-07 2021-08-12 University Of Rochester Ribozyme-mediated rna assembly and expression
WO2023064895A1 (en) * 2021-10-15 2023-04-20 The Broad Institute, Inc. Rna-guided trans-splicing of rna

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
FENYONG LIU ET AL: "Inhibition of viral gene expression by the catalytic RNA subunlt of RNase P from Escherichia coli", GENES & DEVELOPMENT, 15 February 1995 (1995-02-15), US, pages 471 - 480, XP055613887, ISSN: 0890-9369, DOI: 10.1101/gad.9.4.471 *
LI SHAOYA ET AL: "Precise gene replacement in rice by RNA transcript-templated homologous recombination", NATURE BIOTECHNOLOGY, NATURE PUBLISHING GROUP US, NEW YORK, vol. 37, no. 4, 18 March 2019 (2019-03-18), pages 445 - 450, XP036900644, ISSN: 1087-0156, [retrieved on 20190318], DOI: 10.1038/S41587-019-0065-7 *
LI XU ET AL: "Empower multiplex cell and tissue-specific CRISPR-mediated gene manipulation with self-cleaving ribozymes and tRNA", NUCLEIC ACIDS RESEARCH, vol. 45, no. 5, 30 October 2016 (2016-10-30), GB, pages e28, XP055515116, ISSN: 0305-1048, DOI: 10.1093/nar/gkw1048 *
ZHAN YONGHAO ET AL: "Enhanced RNA knockdown efficiency with engineered fusion guide RNAs that function with both CRISPR-CasRx and hammerhead ribozyme", GENOME BIOLOGY, vol. 24, no. 1, 17 January 2023 (2023-01-17), London, UK, XP093275983, ISSN: 1474-760X, DOI: 10.1186/s13059-023-02852-w *

Also Published As

Publication number Publication date
WO2025171380A2 (en) 2025-08-14

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