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WO2012007848A3 - Meganuclease variants cleaving a dna target sequence in the was gene and uses thereof - Google Patents

Meganuclease variants cleaving a dna target sequence in the was gene and uses thereof Download PDF

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Publication number
WO2012007848A3
WO2012007848A3 PCT/IB2011/002408 IB2011002408W WO2012007848A3 WO 2012007848 A3 WO2012007848 A3 WO 2012007848A3 IB 2011002408 W IB2011002408 W IB 2011002408W WO 2012007848 A3 WO2012007848 A3 WO 2012007848A3
Authority
WO
WIPO (PCT)
Prior art keywords
gene
dna target
meganuclease variants
target sequence
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/IB2011/002408
Other languages
French (fr)
Other versions
WO2012007848A2 (en
Inventor
Frederic Cedrone
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
Publication of WO2012007848A2 publication Critical patent/WO2012007848A2/en
Publication of WO2012007848A3 publication Critical patent/WO2012007848A3/en
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

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
    • 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 [RNase]; Deoxyribonucleases [DNase]
    • 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
    • C12N15/87—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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  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

Meganuclease variants cleaving DNA target sequences of the WAS gene, vectors encoding such variants, and cells expressing them. Methods of using meganuclease variants recognizing WAS gene sequences for modifying the WAS gene sequence or for modulating WAS gene, including gene correction, gene insertion, exon knock-in and gene inactivation.
PCT/IB2011/002408 2010-07-16 2011-07-18 Meganuclease variants cleaving a dna target sequence in the was gene and uses thereof Ceased WO2012007848A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US36509610P 2010-07-16 2010-07-16
US61/365,096 2010-07-16

Publications (2)

Publication Number Publication Date
WO2012007848A2 WO2012007848A2 (en) 2012-01-19
WO2012007848A3 true WO2012007848A3 (en) 2012-04-19

Family

ID=44999807

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/IB2011/002408 Ceased WO2012007848A2 (en) 2010-07-16 2011-07-18 Meganuclease variants cleaving a dna target sequence in the was gene and uses thereof

Country Status (1)

Country Link
WO (1) WO2012007848A2 (en)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3137896A1 (en) * 2019-04-24 2020-10-29 Seattle Children's Hospital D/B/A Seattle Children's Research Institute Wiskott-aldrich syndrome gene homing endonuclease variants, compositions, and methods of use
CN111808859B (en) * 2020-07-13 2022-09-13 中国科学院广州生物医药与健康研究院 gRNA of WAS gene and application thereof

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2010026443A1 (en) * 2008-09-08 2010-03-11 Cellectis Meganuclease variants cleaving a dna target sequence from a glutamine synthetase gene and uses thereof

Family Cites Families (19)

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Publication number Priority date Publication date Assignee Title
US4179337A (en) 1973-07-20 1979-12-18 Davis Frank F Non-immunogenic polypeptides
US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
US5006333A (en) 1987-08-03 1991-04-09 Ddi Pharmaceuticals, Inc. Conjugates of superoxide dismutase coupled to high molecular weight polyalkylene glycols
WO2009095742A1 (en) 2008-01-31 2009-08-06 Cellectis New i-crei derived single-chain meganuclease and uses thereof
US8206965B2 (en) 2002-03-15 2012-06-26 Cellectis S.A. Hybrid and single chain meganucleases and use thereof
WO2004031346A2 (en) 2002-09-06 2004-04-15 Fred Hutchinson Cancer Research Center Methods and compositions concerning designed highly-specific nucleic acid binding proteins
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.
WO2007034262A1 (en) 2005-09-19 2007-03-29 Cellectis Heterodimeric meganucleases and use thereof
WO2006097784A1 (en) 2005-03-15 2006-09-21 Cellectis I-crei meganuclease variants with modified specificity, method of preparation and uses thereof
AU2006224248B2 (en) 2005-03-15 2011-01-06 Cellectis I-Crei meganuclease variants with modified specificity, method of preparation and uses thereof
WO2007060495A1 (en) 2005-10-25 2007-05-31 Cellectis I-crei homing endonuclease variants having novel cleavage specificity and use thereof
WO2007049095A1 (en) 2005-10-25 2007-05-03 Cellectis Laglidadg homing endonuclease variants having mutations in two functional subdomains and use thereof
WO2007093836A1 (en) 2006-02-13 2007-08-23 Cellectis Meganuclease variants cleaving a dna target sequence from a xp gene and uses thereof
WO2008010009A1 (en) 2006-07-18 2008-01-24 Cellectis Meganuclease variants cleaving a dna target sequence from a rag gene and uses thereof
BRPI0718747A2 (en) 2006-11-14 2013-12-03 Cellectis MEGANUCLEASE VARIANTS KEYING ONE OR SEQUENCE DNA TARGET FROM THE HPRT GENE AND USES THEREOF.
WO2008102199A1 (en) 2007-02-20 2008-08-28 Cellectis Meganuclease variants cleaving a dna target sequence from the beta-2-microglobulin gene and uses thereof
WO2008149176A1 (en) 2007-06-06 2008-12-11 Cellectis Meganuclease variants cleaving a dna target sequence from the mouse rosa26 locus and uses thereof
WO2009013559A1 (en) 2007-07-23 2009-01-29 Cellectis Meganuclease variants cleaving a dna target sequence from the human hemoglobin beta gene and uses thereof
WO2009019528A1 (en) 2007-08-03 2009-02-12 Cellectis Meganuclease variants cleaving a dna target sequence from the human interleukin-2 receptor gamma chain gene and uses thereof

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2010026443A1 (en) * 2008-09-08 2010-03-11 Cellectis Meganuclease variants cleaving a dna target sequence from a glutamine synthetase gene and uses thereof

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
ARNOULD ET AL: "Engineering of Large Numbers of Highly Specific Homing Endonucleases that Induce Recombination on Novel DNA Targets", JOURNAL OF MOLECULAR BIOLOGY, ACADEMIC PRESS, UNITED KINGDOM, vol. 355, no. 3, 20 January 2006 (2006-01-20), pages 443 - 458, XP005206991, ISSN: 0022-2836, DOI: 10.1016/J.JMB.2005.10.065 *
CHAPDELAINE P ET AL: "Meganucleases can restore the reading frame of a mutated dystrophin", GENE THERAPY, MACMILLAN PRESS LTD., BASINGSTOKE, GB, vol. 17, no. 7, 1 July 2010 (2010-07-01), pages 846 - 858, XP002610887, ISSN: 0969-7128, [retrieved on 20100415] *
MARANGONI FRANCESCO ET AL: "Evidence for long-term efficacy and safety of gene therapy for Wiskott-Aldrich syndrome in preclinical models.", MOLECULAR THERAPY : THE JOURNAL OF THE AMERICAN SOCIETY OF GENE THERAPY JUN 2009 LNKD- PUBMED:19259069, vol. 17, no. 6, June 2009 (2009-06-01), pages 1073 - 1082, XP002669501, ISSN: 1525-0024 *

Also Published As

Publication number Publication date
WO2012007848A2 (en) 2012-01-19

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