WO2012010976A3 - Meganuclease variants cleaving a dna target sequence in the tert gene and uses thereof - Google Patents

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

Info

Publication number
WO2012010976A3
WO2012010976A3 PCT/IB2011/002403 IB2011002403W WO2012010976A3 WO 2012010976 A3 WO2012010976 A3 WO 2012010976A3 IB 2011002403 W IB2011002403 W IB 2011002403W WO 2012010976 A3 WO2012010976 A3 WO 2012010976A3
Authority
WO
WIPO (PCT)
Prior art keywords
gene
tert gene
dna target
tert
meganuclease variants
Prior art date
Application number
PCT/IB2011/002403
Other languages
French (fr)
Other versions
WO2012010976A2 (en
Inventor
Frederic Paques
David Sourdive
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
Publication of WO2012010976A2 publication Critical patent/WO2012010976A2/en
Publication of WO2012010976A3 publication Critical patent/WO2012010976A3/en

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 RNAses, DNAses
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor

Abstract

Meganuclease variants cleaving DNA target sequences of the TERT gene, vectors encoding such variants, and cells expressing them. Methods of using meganuclease variants recognizing TERT gene sequences for modifying the TERT gene sequence, including gene correction and gene inactivation, or for incorporating a gene of interest or therapeutic gene using the TERT gene as a landing pad and a safe harbor locus.
PCT/IB2011/002403 2010-07-15 2011-07-13 Meganuclease variants cleaving a dna target sequence in the tert gene and uses thereof WO2012010976A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US36470010P 2010-07-15 2010-07-15
US61/364,700 2010-07-15

Publications (2)

Publication Number Publication Date
WO2012010976A2 WO2012010976A2 (en) 2012-01-26
WO2012010976A3 true WO2012010976A3 (en) 2012-08-02

Family

ID=44993622

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/IB2011/002403 WO2012010976A2 (en) 2010-07-15 2011-07-13 Meganuclease variants cleaving a dna target sequence in the tert gene and uses thereof

Country Status (1)

Country Link
WO (1) WO2012010976A2 (en)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN102676518B (en) * 2012-02-27 2013-09-18 华侨大学 Novel anti-tumor amiRNA (artificial micro Ribonucleic Acid) sequence and application thereof
US20150104873A1 (en) * 2012-07-11 2015-04-16 University of Nevada, Las Vegas Genome Surgery with Paired, Permeant Endonuclease Construct
KR102630014B1 (en) 2014-10-01 2024-01-25 더 제너럴 하스피탈 코포레이션 Methods for increasing efficiency of nuclease-induced homology-directed repair
WO2016171625A1 (en) * 2015-04-22 2016-10-27 National University Of Singapore Targeting telomerase for cell therapy
ES2953925T3 (en) * 2015-11-04 2023-11-17 Fate Therapeutics Inc Genomic engineering of pluripotent cells

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2006097784A1 (en) * 2005-03-15 2006-09-21 Cellectis I-crei meganuclease variants with modified specificity, method of preparation 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
WO2009095793A1 (en) * 2008-01-31 2009-08-06 Cellectis New i-crei derived single-chain meganuclease and uses thereof
WO2010136981A2 (en) * 2009-05-26 2010-12-02 Cellectis Meganuclease variants cleaving the genome of a pathogenic non-integrating virus and uses thereof
WO2012006440A2 (en) * 2010-07-07 2012-01-12 Cellular Dynamics International, Inc. Endothelial cell production by programming
WO2012004671A2 (en) * 2010-07-07 2012-01-12 Cellectis Meganucleases variants cleaving a dna target sequence in the nanog gene and uses thereof

Family Cites Families (18)

* Cited by examiner, † Cited by third party
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
FR2646438B1 (en) 1989-03-20 2007-11-02 Pasteur Institut A METHOD FOR SPECIFIC REPLACEMENT OF A COPY OF A GENE PRESENT IN THE RECEIVER GENOME BY INTEGRATION OF A GENE DIFFERENT FROM THAT OR INTEGRATION
US5792632A (en) 1992-05-05 1998-08-11 Institut Pasteur Nucleotide sequence encoding the enzyme I-SceI and the uses thereof
JP2005520519A (en) 2002-03-15 2005-07-14 セレクティス Hybrid and single chain meganucleases and uses thereof
AU2003290518A1 (en) 2002-09-06 2004-04-23 Fred Hutchinson Cancer Research Center Methods and compositions concerning designed highly-specific nucleic acid binding proteins
EP2559759A1 (en) 2003-01-28 2013-02-20 Cellectis Custom-made meganuclease and use thereof
ES2347684T3 (en) 2005-03-15 2010-11-03 Cellectis VARIANTS OF MEGANUCLEASE I-CREI WITH MODIFY SPECIFICITY, METHOD OF PREPARATION AND USES OF THE SAME.
WO2007034262A1 (en) 2005-09-19 2007-03-29 Cellectis Heterodimeric meganucleases 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
WO2007060495A1 (en) 2005-10-25 2007-05-31 Cellectis I-crei homing endonuclease variants having novel cleavage specificity 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
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 (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2006097784A1 (en) * 2005-03-15 2006-09-21 Cellectis I-crei meganuclease variants with modified specificity, method of preparation 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
WO2009095793A1 (en) * 2008-01-31 2009-08-06 Cellectis New i-crei derived single-chain meganuclease and uses thereof
WO2010136981A2 (en) * 2009-05-26 2010-12-02 Cellectis Meganuclease variants cleaving the genome of a pathogenic non-integrating virus and uses thereof
WO2012006440A2 (en) * 2010-07-07 2012-01-12 Cellular Dynamics International, Inc. Endothelial cell production by programming
WO2012004671A2 (en) * 2010-07-07 2012-01-12 Cellectis Meganucleases variants cleaving a dna target sequence in the nanog gene and uses thereof

Non-Patent Citations (7)

* 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, ACADEMIC PRESS, UNITED KINGDOM, vol. 355, no. 3, 20 January 2006 (2006-01-20), pages 443 - 458, XP024950505, ISSN: 0022-2836, [retrieved on 20060120], DOI: 10.1016/J.JMB.2005.10.065 *
DIRK HOCKEMEYER ET AL: "Genetic engineering of human pluripotent cells using TALE nucleases", NATURE BIOTECHNOLOGY, vol. 29, no. 8, 1 August 2011 (2011-08-01), pages 731 - 734, XP055018244, ISSN: 1087-0156, DOI: 10.1038/nbt.1927 *
GALETTO ROMAN ET AL: "Targeted approaches for gene therapy and the emergence of engineered meganucleases", EXPERT OPINION ON BIOLOGICAL THERAPY, INFORMA HEALTHCARE, UK, vol. 9, no. 10, 1 October 2009 (2009-10-01), pages 1289 - 1303, XP009137203, ISSN: 1744-7682, DOI: 10.1517/14712590903213669 *
MICHELLE CHRISTIAN ET AL: "Targeting DNA Double-Strand Breaks with TAL Effector Nucleases", GENETICS, GENETICS SOCIETY OF AMERICA, AUSTIN, TX, US, vol. 186, no. 2, 1 October 2010 (2010-10-01), pages 757 - 761, XP002632806, ISSN: 0016-6731, [retrieved on 20100726], DOI: 10.1534/GENETICS.110.120717 *
NOEMI FUSAKI ET AL: "Efficient induction of transgene-free human pluripotent stem cells using a vector based on Sendai virus, an RNA virus that does not integrate into the host genome", PROCEEDINGS OF THE JAPAN ACADEMY. SERIES B, PHYSICAL ANDBIOLOGICAL SCIENCES, TOKYO, JP, vol. 85, no. 8, 1 October 2009 (2009-10-01), pages 348 - 362, XP002663242, ISSN: 0386-2208, DOI: DOI:10.2183/PJAB.85.348 *
SCHAMBACH AXEL ET AL: "Generation and genetic modification of induced pluripotent stem cells", EXPERT OPINION ON BIOLOGICAL THERAPY, INFORMA HEALTHCARE, UK, vol. 10, no. 7, 1 July 2010 (2010-07-01), pages 1089 - 1103, XP009153949, ISSN: 1744-7682, DOI: 10.1517/14712598.2010.496775 *
SUNEET AGARWAL ET AL: "Telomere elongation in induced pluripotent stem cells from dyskeratosis congenita patients", NATURE, vol. 464, no. 7286, 11 March 2010 (2010-03-11), pages 292 - 296, XP055018279, ISSN: 0028-0836, DOI: 10.1038/nature08792 *

Also Published As

Publication number Publication date
WO2012010976A2 (en) 2012-01-26

Similar Documents

Publication Publication Date Title
AU2016202426A1 (en) Vectors conditionally expressing therapeutic proteins, host cells comprising the vectors, and uses thereof
HK1250518A1 (en) Vectors conditionally expressing therapeutic proteins, host cells comprising the vectors, and uses thereof
WO2013063383A3 (en) Vectors encoding rod-derived cone viability factor
WO2014022702A3 (en) Methods and compositions for controlling gene expression by rna processing
WO2014011901A3 (en) Methods and compositions for delivery of biologics
WO2010107493A3 (en) Modification of cxcr4 using engineered zinc finger proteins
WO2012146171A9 (en) Polypeptides having cellulolytic enhancing activity and polynucleotides encoding same
MX2012011774A (en) Polypeptides having glucoamylase activity and polynucleotides encoding same.
WO2013164793A3 (en) Viral vectors for the treatment of retinal dystrophy
MX2013007997A (en) Polypeptides having cellulolytic enhancing activity and polynucleotides encoding same.
WO2012112578A3 (en) Improved aav8 vector with enhanced functional activity and methods of use thereof
WO2012048099A3 (en) Nanoparticle-loaded cells
SG10201606784QA (en) Nucleic acid comprising or coding for a histone stem-loop and a poly(a) sequence or a polyadenylation signal for increasing the expression of an encoded therapeutic protein
WO2012042386A3 (en) Mannosidases capable of uncapping mannose-1-phospho-6-mannose linkages and demannosylating phosphorylated n-glycans and methods of facilitating mammalian cellular uptake of glycoproteins
WO2013009627A3 (en) Factor viii chimeric and hybrid polypeptides, and methods of use thereof
WO2011094358A9 (en) Influenza nucleic acid molecules and vaccines made therefrom
WO2010120514A3 (en) Antigen-binding proteins comprising recombinant protein scaffolds
WO2013170170A3 (en) Compositions and methods for gene therapy
WO2012170431A3 (en) Improved geneswitch systems
WO2008122811A3 (en) Adenoviral vectors encoding a pathogen or tumour antigen
EP4241854A3 (en) Lysosomal storage disease enzyme
WO2010122367A3 (en) Meganuclease variants cleaving the genomic insertion of a virus and uses thereof
WO2012004671A3 (en) Meganucleases variants cleaving a dna target sequence in the nanog gene and uses thereof
WO2012010976A3 (en) Meganuclease variants cleaving a dna target sequence in the tert gene and uses thereof
WO2012160333A3 (en) Recombinant yeast expressing human h- ferritin

Legal Events

Date Code Title Description
121 Ep: the epo has been informed by wipo that ep was designated in this application

Ref document number: 11784769

Country of ref document: EP

Kind code of ref document: A2

NENP Non-entry into the national phase

Ref country code: DE

32PN Ep: public notification in the ep bulletin as address of the adressee cannot be established

Free format text: NOTING OF LOSS OF RIGHTS PURSUANT TO RULE 112(1) EPC

122 Ep: pct application non-entry in european phase

Ref document number: 11784769

Country of ref document: EP

Kind code of ref document: A2