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
Application number
PCT/IB2009/006039
Other languages
French (fr)
Other versions
WO2010136841A2 (en
Inventor
André CHOULIKA
Julianne Smith
Original Assignee
Cellectis
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 filed Critical Cellectis
Priority to PCT/IB2009/006039 priority Critical patent/WO2010136841A2/en
Priority to PCT/IB2010/052340 priority patent/WO2010136981A2/en
Priority to US13/322,266 priority patent/US20120171191A1/en
Priority to EP10726296A priority patent/EP2435560A2/en
Publication of WO2010136841A2 publication Critical patent/WO2010136841A2/en
Publication of WO2010136841A3 publication Critical patent/WO2010136841A3/en
Priority to US14/744,668 priority patent/US20150315557A1/en

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    • 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
    • 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 RNAses, DNAses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/20—Antivirals for DNA viruses
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal 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 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
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
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
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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Application Number Title Priority Date Filing Date
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

Country Status (1)

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WO (1) WO2010136841A2 (en)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3526323B1 (en) 2016-10-14 2023-03-29 Precision Biosciences, Inc. Engineered meganucleases specific for recognition sequences in the hepatitis b virus genome
TW201945388A (en) 2018-04-12 2019-12-01 美商精密生物科學公司 Optimized engineered meganucleases having specificity for a recognition sequence in the hepatitis B virus genome
EP4069729A1 (en) 2019-12-06 2022-10-12 Precision BioSciences, Inc. Optimized engineered meganucleases having specificity for a recognition sequence in the hepatitis b virus genome

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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