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WO2010136841A3 - Meganuclease variants cleaving the genome of a non-genomically integrating virus and uses thereof - Google Patents

Meganuclease variants cleaving the genome of a non-genomically integrating virus and uses thereof Download PDF

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Publication number
WO2010136841A3
WO2010136841A3 PCT/IB2009/006039 IB2009006039W WO2010136841A3 WO 2010136841 A3 WO2010136841 A3 WO 2010136841A3 IB 2009006039 W IB2009006039 W IB 2009006039W WO 2010136841 A3 WO2010136841 A3 WO 2010136841A3
Authority
WO
WIPO (PCT)
Prior art keywords
genome
variants
integrating virus
meganuclease variants
variants cleaving
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.)
Ceased
Application number
PCT/IB2009/006039
Other languages
French (fr)
Other versions
WO2010136841A2 (en
Inventor
André CHOULIKA
Julianne Smith
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.)
Cellectis SA
Original Assignee
Cellectis SA
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 Cellectis SA filed Critical Cellectis SA
Priority to PCT/IB2009/006039 priority Critical patent/WO2010136841A2/en
Priority to US13/322,266 priority patent/US20120171191A1/en
Priority to EP10726296A priority patent/EP2435560A2/en
Priority to PCT/IB2010/052340 priority patent/WO2010136981A2/en
Publication of WO2010136841A2 publication Critical patent/WO2010136841A2/en
Publication of WO2010136841A3 publication Critical patent/WO2010136841A3/en
Anticipated expiration legal-status Critical
Priority to US14/744,668 priority patent/US20150315557A1/en
Ceased legal-status Critical Current

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Classifications

    • 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]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • A61P31/20Antivirals for DNA viruses
    • 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
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102Mutagenizing nucleic acids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Virology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Oncology (AREA)
  • Communicable Diseases (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)

Abstract

The present invention relates to variants of the meganuclease l-CreI which are characterized in that they are able to cleave a DNA target sequen from a non-integrating virus (NIV) genome. The present invention also relat to rational methods to generate such l-Crel variants and the use of the variants as research and diagnostic reagents, as well as medicamen comprising these I-Crel variants for the treatment of NIV infections.
PCT/IB2009/006039 2009-05-26 2009-05-26 Meganuclease variants cleaving the genome of a non-genomically integrating virus and uses thereof Ceased WO2010136841A2 (en)

Priority Applications (5)

Application Number Priority Date Filing Date Title
PCT/IB2009/006039 WO2010136841A2 (en) 2009-05-26 2009-05-26 Meganuclease variants cleaving the genome of a non-genomically integrating virus and uses thereof
US13/322,266 US20120171191A1 (en) 2009-05-26 2010-05-26 Meganuclease variants cleaving the genome of a pathogenic non-integrating virus and uses thereof
EP10726296A EP2435560A2 (en) 2009-05-26 2010-05-26 Meganuclease variants cleaving the genome of a pathogenic non-integrating virus and uses thereof
PCT/IB2010/052340 WO2010136981A2 (en) 2009-05-26 2010-05-26 Meganuclease variants cleaving the genome of a pathogenic non-integrating virus and uses thereof
US14/744,668 US20150315557A1 (en) 2009-05-26 2015-06-19 Meganuclease variants cleaving the genome of a pathogenic non-integrating virus and uses thereof

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
PCT/IB2009/006039 WO2010136841A2 (en) 2009-05-26 2009-05-26 Meganuclease variants cleaving the genome of a non-genomically integrating virus and uses thereof

Publications (2)

Publication Number Publication Date
WO2010136841A2 WO2010136841A2 (en) 2010-12-02
WO2010136841A3 true WO2010136841A3 (en) 2011-03-17

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/IB2009/006039 Ceased WO2010136841A2 (en) 2009-05-26 2009-05-26 Meganuclease variants cleaving the genome of a non-genomically integrating virus and uses thereof

Country Status (1)

Country Link
WO (1) WO2010136841A2 (en)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018071849A2 (en) 2016-10-14 2018-04-19 Precision Biosciences, Inc. Engineered meganucleases specific for recognition sequences in the hepatitis b virus genome
US11142750B2 (en) 2018-04-12 2021-10-12 Precision Biosciences, Inc. Optimized engineered meganucleases having specificity for a recognition sequence in the Hepatitis B virus genome
ES3022990T3 (en) 2019-12-06 2025-05-29 Prec Biosciences Inc Optimized engineered meganucleases having specificity for a recognition sequence in the hepatitis b virus genome
US12390538B2 (en) 2023-05-15 2025-08-19 Nchroma Bio, Inc. Compositions and methods for epigenetic regulation of HBV gene expression

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2004067753A2 (en) * 2003-01-28 2004-08-12 Cellectis Use of meganucleases for inducing homologous recombination ex vivo and in toto in vertebrate somatic tissues and application thereof.
WO2007047859A2 (en) * 2005-10-18 2007-04-26 Precision Biosciences Rationally-designed meganucleases with altered sequence specificity and dna-binding affinity
WO2007057781A2 (en) * 2005-10-25 2007-05-24 Cellectis Laglidadg homing endonuclease variants having mutations in two functional subdomains and use thereof.
WO2008059382A2 (en) * 2006-11-14 2008-05-22 Cellectis Meganuclease variants cleaving a dna target sequence from the hprt gene and uses thereof
WO2008102274A2 (en) * 2007-02-20 2008-08-28 Cellectis Meganuclease variants cleaving a dna target sequence from the beta-2-microglobulin gene and uses thereof
WO2009013622A2 (en) * 2007-07-23 2009-01-29 Cellectis Meganuclease variants cleaving a dna target sequence from the human hemoglobin beta gene and uses thereof

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2004067753A2 (en) * 2003-01-28 2004-08-12 Cellectis Use of meganucleases for inducing homologous recombination ex vivo and in toto in vertebrate somatic tissues and application thereof.
WO2007047859A2 (en) * 2005-10-18 2007-04-26 Precision Biosciences Rationally-designed meganucleases with altered sequence specificity and dna-binding affinity
WO2007057781A2 (en) * 2005-10-25 2007-05-24 Cellectis Laglidadg homing endonuclease variants having mutations in two functional subdomains and use thereof.
WO2008059382A2 (en) * 2006-11-14 2008-05-22 Cellectis Meganuclease variants cleaving a dna target sequence from the hprt gene and uses thereof
WO2008102274A2 (en) * 2007-02-20 2008-08-28 Cellectis Meganuclease variants cleaving a dna target sequence from the beta-2-microglobulin gene and uses thereof
WO2009013622A2 (en) * 2007-07-23 2009-01-29 Cellectis Meganuclease variants cleaving a dna target sequence from the human hemoglobin beta gene and uses thereof

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
ARNOULD S ET AL: "Engineering of Large Numbers of Highly Specific Homing Endonucleases that Induce Recombination on Novel DNA Targets", JOURNAL OF MOLECULAR BIOLOGY, LONDON, GB, vol. 355, no. 3, 20 January 2006 (2006-01-20), pages 443 - 458, XP024950505, ISSN: 0022-2836, [retrieved on 20060120] *
CHEVALIER B ET AL: "Flexible DNA Target Site Recognition by Divergent Homing Endonuclease Isoschizomers I-CreI and I-MsoI", JOURNAL OF MOLECULAR BIOLOGY, LONDON, GB, vol. 329, no. 2, 30 May 2003 (2003-05-30), pages 253 - 269, XP004454255, ISSN: 0022-2836 *
PÂQUES FRÉDÉRIC ET AL: "Meganucleases and DNA double-strand break-induced recombination: perspectives for gene therapy", CURRENT GENE THERAPY, BENTHAM SCIENCE PUBLISHERS LTD, NL, vol. 7, no. 1, 1 February 2007 (2007-02-01), pages 49 - 66, XP002493370, ISSN: 1566-5232 *
ROSEN LAURA E ET AL: "Homing endonuclease I-CreI derivatives with novel DNA target specificities", NUCLEIC ACIDS RESEARCH, vol. 34, no. 17, October 2006 (2006-10-01), pages 4791 - 4800, XP002554264, ISSN: 0305-1048 *
SMITH JULIANNE ET AL: "A combinatorial approach to create artificial homing endonucleases cleaving chosen sequences", NUCLEIC ACIDS RESEARCH, OXFORD UNIVERSITY PRESS, SURREY, GB, vol. 34, no. 22, 27 November 2006 (2006-11-27), pages E149 - 1, XP002457876, ISSN: 0305-1048 *

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Publication number Publication date
WO2010136841A2 (en) 2010-12-02

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