CELLECTIS Patent applications |
Patent application number | Title | Published |
20160120905 | METHODS FOR ENGINEERING ALLOGENEIC AND HIGHLY ACTIVE T CELL FOR IMMUNOTHERAPHY - The present invention relates to methods for developing engineered T-cells for immunotherapy that are non-alloreactive. The present invention relates to methods for modifying T-cells by inactivating both genes encoding T-cell receptor and an immune checkpoint gene to unleash the potential of the immune response. This method involves the use of specific rare cutting endonucleases, in particular TALE-nucleases (TAL effector endonuclease) and polynucleotides encoding such polypeptides, to precisely target a selection of key genes in T-cells, which are available from donors or from culture of primary cells. The invention opens the way to standard and affordable adoptive immunotherapy strategies for treating cancer and viral infections. | 05-05-2016 |
20150315557 | MEGANUCLEASE VARIANTS CLEAVING THE GENOME OF A PATHOGENIC NON-INTEGRATING VIRUS AND USES THEREOF - An I-CreI variant, wherein at least one of the two I-Cre1 monomers has at least two substitutions, one in each of the two functional subdomains of the LAGLIDADG core domain situated from positions 26 to 40 and 44 to 77 of I-CreI, said variant being able to cleave a DNA target sequence from the genome of a non-integrating virus, in particular herpes simplex virus (HSV) or Hepatitis B virus (HBV) for use in genome engineering and for in vivo and ex vivo (gene cell therapy) genome therapy as well as the treatment of a virus infection. | 11-05-2015 |
20150225465 | NEW MODULAR BASE-SPECIFIC NUCLEIC ACID BINDING DOMAINS FROM BURKHOLDERIA RHIZOXINICA PROTEINS - The present invention concerns new modular base-per-base specific nucleic acid binding domains (MBBBD) derived from newly identified proteins from the bacterial endosymbiont | 08-13-2015 |
20150203817 | USE OF PRE T ALPHA OR FUNCTIONAL VARIANT THEREOF FOR EXPANDING TCR ALPHA DEFICIENT T CELLS - A method of expanding TCRalpha deficient T-cells by expressing pTalpha or functional variants thereof into said cells, thereby restoring a functional CD3 complex. This method is particularly useful to enhance the efficiency of immunotherapy using primary T-cells from donors. This method involves the use of pTalpha or functional variants thereof and polynucleotides encoding such polypeptides to expand TCRalpha deficient T-cells. Such engineered cells can be obtained by using specific rare-cutting endonuclease, preferably TALE-nucleases. The use of Chimeric Antigen Receptor (CAR), especially multi-chain CAR, in such engineered cells to target malignant or infected cells. The invention opens the way to standard and affordable adoptive immunotherapy strategies for treating cancer and viral infections. | 07-23-2015 |
20150017136 | METHODS FOR ENGINEERING ALLOGENEIC AND HIGHLY ACTIVE T CELL FOR IMMUNOTHERAPY - The present invention relates to methods for developing engineered T-cells for immunotherapy that are non-alloreactive. The present invention relates to methods for modifying T-cells by inactivating both genes encoding T-cell receptor and an immune checkpoint gene to unleash the potential of the immune response. This method involves the use of specific rare cutting endonucleases, in particular TALE-nucleases (TAL effector endonuclease) and polynucleotides encoding such polypeptides, to precisely target a selection of key genes in T-cells, which are available from donors or from culture of primary cells. The invention opens the way to standard and affordable adoptive immunotherapy strategies for treating cancer and viral infections. | 01-15-2015 |
20140370558 | MODIFYING SOYBEAN OIL COMPOSITION THROUGH TARGETED KNOCKOUT OF THE FAD2-1A/1B GENES - Materials and methods are provided for making soybean varieties that have altered oil composition as a result of mutations in the FAD2-1A and FAD2-1B genes. | 12-18-2014 |
20140234975 | METHOD FOR INCREASING THE EFFICIENCY OF DOUBLE-STRAND-BREAK INDUCED MUTAGENESIS - The present invention relates to a method for increasing double-strand-break induced mutagenesis at a genomic locus of interest in a cell, thereby giving new tools for genome engineering, including therapeutic applications and cell line engineering. More specifically, the present invention concerns the combined use of TALEN or meganucleases with TREX2, especially under the form of single-chain proteins. | 08-21-2014 |
20140178942 | MEGANUCLEASE VARIANTS CLEAVING AT LEAST ONE TARGET IN THE GENOME OF A RETROVIRUS AND USES THEREOF - Meganuclease variants which cleave at least one target in the provirus of a retrovirus and in particular which cleave the genomic insertion of the provirus. The present invention particular relates to meganuclease variants which cleave the provirus of the Human Immunodeficiency Virus genome following genomic insertion. Vector encoding such variants, as well as to a cell or multi-cellular organism modified by such a vector and use of said meganuclease variants and derived products for genome engineering and for in vivo and ex vivo (gene cell therapy) genome therapy. | 06-26-2014 |
20140178561 | POTATOES WITH REDUCED COLD-INDUCED SWEETENING - Materials and methods are provided for making plants (e.g., | 06-26-2014 |
20140134142 | Multi-Chain Chimeric Antigen Receptor and Uses Thereof - The present invention relates to a new generation of chimeric antigen receptors (CAR) referred to as multi-chain CARs. Such CARs, which aim to redirect immune cell specificity and reactivity toward a selected target exploiting the ligand-binding domain properties, comprise separate extracellular ligand binding and signaling domains in different transmembrane polypeptides. The signaling domains are designed to assemble in juxtamembrane position, which forms flexible architecture closer to natural receptors, that confers optimal signal transduction. The invention encompasses the polynucleotides, vectors encoding said multi-chain CAR and the isolated cells expressing them at their surface, in particularly for their use in immunotherapy. The invention opens the way to efficient adoptive immunotherapy strategies for treating cancer and viral infections. | 05-15-2014 |
20140121115 | CUSTOM-MADE MEGANUCLEASE AND USE THEREOF - New rare-cutting endonucleases, also called custom-made meganucleases, which recognize and cleave a specific nucleotide sequence, derived polynucleotide sequences, recombinant vector cell, animal, or plant comprising said polynucleotide sequences, process for producing said rare-cutting endonucleases and any use thereof, more particularly, for genetic engineering, antiviral therapy and gene therapy. | 05-01-2014 |
20140115726 | NEW TALE-PROTEIN SCAFFOLDS AND USES THEREOF - The present invention relates to new Transcription Activator-Like Effector proteins and more particularly new Transcription Activator-Like Effector Nucleases (TALENs) that can efficiently target and process nucleic acids. The present invention also concerns methods to use these new Transcription Activator-Like Effector proteins. The present invention also relates to vectors, compositions and kits in which Transcription Activator-Like Effector proteins of the present invention are used. | 04-24-2014 |
20140112904 | METHOD FOR ENHANCING THE CLEAVAGE ACTIVITY OF I-CREI DERIVED MEGANUCLEASES - A method for enhancing the cleavage activity of an I-CreI derived meganuclease, comprising the site-specific mutation of at least one amino acid residue which is selected in the group consisting of: the glycine at position 19, the phenylalanine at position 54, the phenylalanine at position 87, the serine at position 79, the valine at position 105 and the isoleucine at position 132 of I-CreI, and its application for the manufacturing of meganuclease cleaving a DNA target of interest, for use in genome therapy (treatment of genetic diseases) and genome engineering (making of transgenic animals, transgenic plants and recombinant cell lines). | 04-24-2014 |
20140038239 | MEGANUCLEASE VARIANTS CLEAVING A DNA TARGET SEQUENCE FROM THE HUMAN HEMOGLOBIN BETA GENE AND USES THEREOF - An I-CreI variant, wherein one of the two I-CreI monomers has at least two substitutions, one in each of the two functional subdomains of the LAGLIDADG core domain situated respectively from positions 26 to 40 and 44 to 77 of I-CreI, said variant being able to cleave a DNA target sequence from the human beta globin gene. Use of said variant and derived products for the prevention and the treatment of pathological conditions caused by a mutation in the human beta globin gene (sickle cell disease, beta-thalassemia). | 02-06-2014 |
20140017731 | MEGANUCLEASE VARIANTS CLEAVING A DNA TARGET SEQUENCE FROM THE HUMAN INTERLEUKIN-2 RECEPTOR GAMMA CHAIN GENE AND USES THEREOF - An I-CreI variant, wherein at least one of the two I-CreI monomers has at least two substitutions, one in each of the two functional subdomains of the LAGLIDADG core domain situated respectively from positions 26 to 40 and 44 to 77 of I-CreI, said variant being able to cleave a DNA target sequence from the human IL2RG gene. Use of said variant and derived products for the prevention and the treatment of X-linked severe combined immunodeficiency. | 01-16-2014 |
20140004608 | MEGANUCLEASE RECOMBINATION SYSTEM | 01-02-2014 |
20130236946 | MEGANUCLEASE VARIANTS CLEAVING A DNA TARGET SEQUENCE FROM THE MOUSE ROSA26 LOCUS AND USES THEREOF - An I-CreI variant, wherein one of the two I-CreI monomers has at least two substitutions, one in each of the two functional subdomains of the LAGLIDADG core domain situated respectively from positions 26 to 40 and 44 to 77 of I-CreI, said variant being able to cleave a DNA target sequence from the mouse ROSA26 locus. Use of said variant and derived products for the engineering of transgenic mice and recombinant mouse cell lines expressing an heterologous protein of interest. | 09-12-2013 |
20130227715 | USE OF ENDONUCLEASES FOR INSERTING TRANSGENES INTO SAFE HARBOR LOCI - The present invention concerns the endonucleases capable of cleaving a target sequence located in a “safe harbor loci”, i.e. a loci allowing safe expression of a transgene. The present invention further concerns the use of such endonucleases for inserting transgenes into a cell, tissue or individual. | 08-29-2013 |
20130209437 | I-CREI HOMING ENDONUCLEASE VARIANTS HAVING NOVEL CLEAVAGE SPECIFICITY AND USE THEREOF - A method for engineering I-CreI homing endonuclease variants able to cleave mutant I-CreI sites having variation in positions ±8 to ±10. A I-CreI homing endonuclease variant obtainable by said method, a vector encoding said variant, a cell, an animal or a plant modified by said vector. Use of said I-CreI endonuclease variant and derived products for genetic engineering, genome therapy and antiviral therapy. | 08-15-2013 |
20130196320 | METHOD FOR IMPROVING CLEAVAGE OF DNA BY ENDONUCLEASE SENSITIVE TO METHYLATION - The present invention concerns novel methods for improving cleavage of DNA by rare-cutting endonucleases, overcoming DNA modification constraints, particularly DNA methylation, thereby giving new tools for genome engineering, particularly to increase the integration efficiency of a transgene into a genome at a predetermined location, including therapeutic applications and cell line engineering. | 08-01-2013 |
20130190385 | METHOD FOR MODULATING DOUBLE-STRAND BREAK-INDUCED HOMOLOGOUS RECOMBINATION - The present invention concerns a method for modulating double-strand break-induced homologous recombination through the identification of effectors that modulate said double-strand break-induced homologous recombination by uses of interfering agents; these agents are capable of modulating double-strand break-induced homologous recombination through their respective actions on said effectors. The present invention also concerns the uses of these effectors and interfering agents and derivatives, respectively, by introducing them in an eukaryotic cell in order to modulate and more particularly to increase double-strand break-induced homologous recombination and gene targeting efficiency. The present invention also relates to specific derivatives of identified effectors and interfering agents, vectors encoding them, compositions and kits comprising such derivatives in order to modulate and more particularly to increase double-strand break-induced homologous recombination and gene targeting efficiency. | 07-25-2013 |
20130189759 | MEGANUCLEASES VARIANTS CLEAVING A DNA TARGET SEQUENCE IN THE NANOG GENE AND USES THEREOF - Meganuclease variants cleaving DNA target sequences of the NANOG gene, vectors encoding such variants, and cells expressing them. Methods of using meganuclease variants recognizing NANOG gene sequences for modifying the NANOG gene sequence or for incorporating a gene of interest or therapeutic gene using the NANOG gene as a landing pad and a safe harbor locus. | 07-25-2013 |
20130183282 | Meganuclease variants cleaving a DNA target sequence from the rhodopsin gene and uses thereof - The invention relates to meganuclease variants which cleave a DNA target sequence from the human Rhodopsin gene (RHO), to vectors encoding such variants, to a cell, an animal or a plant modified by such vectors and to the use of these meganuclease variants and products derived therefrom for genome therapy, ex vivo (gene cell therapy) and genome engineering including therapeutic applications and cell line engineering. | 07-18-2013 |
20130149786 | I-CREI VARIANTS WITH NEW SPECIFICITY AND METHODS OF THEIR GENERATION - The present invention relates to 1-Cre1 variants which can in particular recognise and cleave DNA targets which do not comprise the same nucleotides at positions ±6 and ±7 which are present in the wild type 1-Cre1 target. The present invention also relates to 1-Cre1 variants which can recognise and cleave targets which do not comprise the wild type nucleotides at positions ±4, ±5, ±6, ±7 and to 1-Cre1 variants with new specificity which can recognise and cleave targets which do not comprise the wild type nucleotides at positions ±4, ±5, ±6, ±7, ±8, ±9 and ±10. | 06-13-2013 |
20130145487 | MEGANUCLEASE VARIANTS CLEAVING A DNA TARGET SEQUENCE FROM THE DYSTROPHIN GENE AND USES THEREOF - The invention relates to meganuclease variants which cleave a DNA target sequence from the human dystrophin gene (DMD), to vectors encoding such variants, to a cell, an animal or a plant modified by such vectors and to the use of these meganuclease variants and products derived therefrom for genome therapy, ex vivo (gene cell therapy) and genome engineering including therapeutic applications and cell line engineering. The invention also relates to the use of meganuclease variants for inserting therapeutic transgenes other than DMD at the dystrophin gene locus, using this locus as a safe harbor locus. The invention also relates to the use of meganuclease variants for using the dystrophin gene locus as a landing pad to insert and express genes of interest. | 06-06-2013 |
20130137644 | CELL PENETRATING PEPTIDE CONJUGATES FOR DELIVERING OF NUCLEIC ACIDS INTO A CELL - The invention provides cell penetrating peptide-nucleic acid conjugates having the formula P-L-N, wherein P is a cell penetrating peptide, N is a nucleic acid, preferably an oligonucleotide and more preferably a siRNA, and L is a hydrophilic polymer, preferably a polyethylene glycol (PEG)-based linker linking P and N together. Compositions, methods of use and methods for producing such conjugates are also disclosed. | 05-30-2013 |
20130059387 | MEGANUCLEASE VARIANTS CLEAVING A DNA TARGET SEQUENCE FROM THE HPRT GENE AND USES THEREOF - A method for inducing a site-specific modification in the HPRT gene, for a non-therapeutic purpose, by contacting a DNA target sequence selected from the group consisting of the sequences SEQ ID NO: 1 to 14 thereby cleaving the DNA target with an I-CreI variant or single-chain derivative having at least one substitution in one of the two functional subdomains of the LAGLIDADG (SEQ ID NO: 153) core domain situated from positions 26 to 40 and 44 to 77 of I-CreI. | 03-07-2013 |
20130045539 | MEGANUCLEASE RECOMBINATION SYSTEM - The invention relates to a set of genetic constructs which comprises at least a first recombinogenic construct (i) with at least two portions homologous to the genomic regions preceding and following the DNA target site of a site specific endonuclease and also comprising both a negative selection and positive selection mark interposed with the homologous portions as well as a region into which a sequence of interest can be cloned adjacent to the positive selection marker; and a second construct (ii, iii or iv) comprising the meganuclease. The present invention also relates to a kit comprising these constructs and methods to use this set of constructs to introduce into the genome of a target cell, tissue or organism a sequence of interest. | 02-21-2013 |
20120301456 | MEGANUCLEASE REAGENTS OF USES THEREOF FOR TREATING GENETIC DISEASES CAUSED BY FRAME SHIFT/NON SENSE MUTATIONS - The present invention relates to a method to treat a genetic disease in an individual caused by at least one frame shift or at least one non sense mutation in the human dystrophin gene comprising at least the step of bringing into contact at least one meganuclease enzyme, which recognizes and cuts a target site in the human dystrophin gene, with the genome of said individual under conditions wherein said at least one meganuclease recognizes and cleaves its target site in the human dystrophin gene. Said method applies also to a set of meganuclease enzymes, which each recognizes and cuts a different target site. The present invention also relates to a kit comprising, at least one meganuclease enzyme as defined above and medicament comprising said meganuclease. | 11-29-2012 |
20120272348 | VIRAL VECTORS ENCODING A DNA REPAIR MATRIX AND CONTAINING A VIRION-ASSOCIATED SITE SPECIFIC MEGANUCLEASE FOR GENE TARGETING - The present invention relates to a fusion protein which comprises at least a functional meganuclease and a viral protein and in particular to fusion protein comprising at least a meganuclease, which recognises and cleaves a specific DNA target sequence and a viral peptide selected from the group Vpr and Vpx or a fragment or derivative thereof; wherein said fusion protein is able to associate with Lentivirus vector particles and following transduction into a host cell recognise and cleave said specific DNA target in vivo. The present Patent Application also relates to a viral particle comprising such a fusion protein and to the use of such fusion proteins and viral particles for gene targeting. | 10-25-2012 |
20120260356 | MEGANUCLEASE VARIANTS CLEAVING AT LEAST ONE TARGET IN THE GENOME OF A RETROVIRUS AND USES THEREOF - Meganuclease variants which cleave at least one target in the provirus of a retrovirus and in particular which cleave the genomic insertion of the provirus. The present invention in particular relates to meganuclease variants which cleave the provirus of the Human Immunodeficiency Virus genome following genomic insertion. Vector encoding such variants, as well as to a cell or multi-cellular organism modified by such a vector and use of said meganuclease variants and derived products for genome engineering and for in vivo and ex vivo (gene cell therapy) genome therapy. | 10-11-2012 |
20120258537 | I-CREI MEGANUCLEASE VARIANTS WITH MODIFIED SPECIFICITY, METHOD OF PREPARATION AND USES THEREOF - Method of preparing I-CreI meganuclease variants having a modified cleavage specificity, variants obtainable by said method and their applications either for cleaving new DNA target or for genetic engineering and genome engineering for non-therapeutic purposes. Nucleic acids encoding said variants, expression cassettes comprising said nucleic acids, vectors comprising said expression cassettes, cells or organisms, plants or animals except humans, transformed by said vectors. | 10-11-2012 |
20120171191 | MEGANUCLEASE VARIANTS CLEAVING THE GENOME OF A PATHOGENIC NON-INTEGRATING VIRUS AND USES THEREOF - An I-CreI variant, wherein at least one of the two 1-Cre1 monomers has at least two substitutions, one in each of the two functional subdomains of the LAGLIDADG core domain situated from positions 26 to 40 and 44 to 77 of I-CreI, said variant being able to cleave a DNA target sequence from the genome of a non-integrating virus, in particular herpes simplex virus (HSV) or Hepatitis B virus (HBV) for use in genome engineering and for in vivo and ex vivo (gene cell therapy) genome therapy as well as the treatment of a virus infection. | 07-05-2012 |
20110263028 | MEGANUCLEASE RECOMBINATION SYSTEM - The invention relates to a set of genetic constructs which allow the efficient and reproducible introduction of a specific nucleotide sequence at a fixed position in the genome by generating a double strand break at a specific position in the genome using a meganuclease and so stimulating a homologous recombination event at this locus between the genomic site and a transfected donor sequence. The present invention also relates to methods using these constructs and to these materials in the form of a kit. | 10-27-2011 |
20110225664 | MEGANUCLEASE VARIANTS CLEAVING A DNA TARGET SEQUENCE FROM A GLUTAMINE SYNTHETASE GENE AND USES THEREOF - An 1-Cre1 variant, wherein one of the two 1-Cre1 monomers has at least two substitutions, one in each of the two functional subdomains of the LAGLIDADG core domain situated respectively from positions 28 to 40 and 44 to 77 of 1-Cre1, said variant being able to cleave a DNA target sequence from the Glutamine Synthetase gene. Use of said variant and derived products for improving expression system for the production of recombinant protein. | 09-15-2011 |
20110207199 | NOVEL METHOD TO GENERATE MEGANUCLEASES WITH ALTERED CHARACTERISTICS - Method to generate and select a meganuclease having at least two altered characteristics in comparison to a parent meganuclease, comprising the steps: a. constructing from a parent meganuclease, a first series of variants which differ from said parent meganuclease by at least one acid amino substitution; b. screening the variants from said first series of step a. and selecting those which have a first altered characteristic; c. constructing from the selected variants of step b. a second series of variants having a least one other amino acid substitution; d. screening the variants from said series of step b. and selecting those which have said first altered characteristic and a second altered characteristic. Polypeptide obtained from said method. | 08-25-2011 |
20110191870 | I-CREI HOMING ENDONUCLEASE VARIANTS HAVING NOVEL CLEAVAGE SPECIFICITY AND USE THEROF - A method for engineering I-CreI homing endonuclease variants able to cleave mutant I-CreI sites having variation in positions ±8 to ±10. A I-CreI homing endonuclease variant obtainable by said method, a vector encoding said variant, a cell, an animal or a plant modified by said vector. Use of said I-CreI endonuclease variant and derived products for genetic engineering, genome therapy and antiviral therapy. | 08-04-2011 |
20110173710 | CHIMERIC MEGANUCLEASE ENZYMES AND USES THEREOF - The current invention relates to polypeptides encoding mutant I-DmoI derivatives with enhanced cleavage activity and altered sequence specificity and uses of these polypeptides. These polypeptides comprise at least the first I-DmoI domain, and the peptide sequence comprises the substitution of at least one of residues 15, 19 and/or 20 as well as at least one of the residues in positions 27, 29, 33, 35, 37, 75, 76, 77, 81 of the first I-DmoI domain. | 07-14-2011 |
20110158974 | Heterodimeric Meganucleases and Use Thereof - Heterodimeric meganuclease comprising two domains of different meganucleases which are in two separate polypeptides, said heterodimeric meganuclease being able to cleave a chimeric DNA target sequence comprising one different half of each parent meganuclease DNA target sequence. | 06-30-2011 |
20110091441 | MEGANUCLEASE VARIANTS CLEAVING A DNA TARGET SEQUENCE FROM THE HUMAN INTERLEUKIN-2 RECEPTOR GAMMA CHAIN GENE AND USES THEREOF - An I-CreI variant, wherein at least one of the two I-CreI monomers has at least two substitutions, one in each of the two functional subdomains of the LAGLIDADG core domain situated respectively from positions 26 to 40 and 44 to 77 of I-CreI, said variant being able to cleave a DNA target sequence from the human IL2RG gene. Use of said variant and derived products for the prevention and the treatment of X-linked severe combined immunodeficiency. | 04-21-2011 |
20110072527 | I-CREI MEGANUCLEASE VARIANTS WITH MODIFIED SPECIFICITY, METHOD OF PREPARATION AND USES THEREOF - Method of preparing I-CreI meganuclease variants having a modified cleavage specificity, variants obtainable by said method and their applications either for cleaving new DNA target or for genetic engineering and genome engineering for non-therapeutic purposes. Nucleic acids encoding said variants, expression cassettes comprising said nucleic acids, vectors comprising said expression cassettes, cells or organisms, plants or animals except humans, transformed by said vectors. | 03-24-2011 |
20110041194 | I-MSOI HOMING ENDONUCLEASE VARIANTS HAVING NOVEL SUBSTRATE SPECIFICITY AND USE THEREOF - An I-MsoI homing endonuclease variant able to cleave mutant I-MsoI sites having variation at positions ±8 to ±10, a vector encoding said variant, a cell, an animal or a plant modified by said vector. Use of said I-MsoI endonuclease variant and derived products for genetic engineering, genome therapy and antiviral therapy. | 02-17-2011 |
20100325745 | MEGANUCLEASE VARIANTS CLEAVING A DNA TARGET SEQUENCE FROM THE MOUSE ROSA26 LOCUS AND USES THEREOF - An I-CreI variant, wherein one of the two I-CreI monomers has at least two substitutions, one in each of the two functional subdomains of the LAGLIDADG (SEQ ID NO: 150) core domain situated respectively from positions 26 to 40 and 44 to 77 of I-CreI, said variant being able to cleave a DNA target sequence from the mouse ROSA26 locus. Use of said variant and derived products for the engineering of transgenic mice and recombinant mouse cell lines expressing an heterologous protein of interest. | 12-23-2010 |
20100229252 | MEGANUCLEASE VARIANTS CLEAVING A DNA TARGET SEQUENCE FROM THE HUMAN HEMOGLOBIN BETA GENE AND USES THEREOF - An I-CreI variant, wherein one of the two I-CreI monomers has at least two substitutions, one in each of the two functional subdomains of the LAGLIDADG core domain situated respectively from positions 26 to 40 and 44 to 77 of I-CreI, said variant being able to cleave a DNA target sequence from the human beta globin gene. Use of said variant and derived products for the prevention and the treatment of pathological conditions caused by a mutation in the human beta globin gene (sickle cell disease, beta-thalassemia). | 09-09-2010 |
20100203031 | METHOD FOR ENHANCING THE CLEAVAGE ACTIVITY OF I-CREI DERIVED MEGANUCLEASES - A method for enhancing the cleavage activity of an I-CreI derived meganuclease, comprising the site-specific mutation of at least one amino acid residue which is selected in the group consisting of: the glycine at position 19, the phenylalanine at position 54, the phenylalanine at position 87, the serine at position 79, the valine at position 105 and the isoleucine at position 132 of I-CreI, and its application for the manufacturing of meganuclease cleaving a DNA target of interest, for use in genome therapy (treatment of genetic diseases) and genome engineering (making of transgenic animals, transgenic plants and recombinant cell lines). | 08-12-2010 |
20100167357 | OBLIGATE HETERODIMER MEGANUCLEASES AND USES THEREOF - An obligate heterodimer meganuclease consisting of a first and a second monomer, deriving from two different homodimeric LAGLIDADG (SEQ ID NO: 66) endonuclease monomers (parent monomers), and having at least one pair of mutations interesting corresponding residues of said parent monomers which make an intermolecular interaction between the two monomers of each parent homodimeric LAGLIDADG (SEQ ID NO: 66) endonuclease, a vector encoding said meganuclease, a cell, an animal or a plant modified by said vector and the use of said meganuclease and derived products for molecular biology, genome engineering and genome therapy. | 07-01-2010 |
20100151556 | HYBRID AND SINGLE CHAIN MEGANUCLEASES AND USE THEREOF - This patent application relates to hybrid and/or single-chain rare-cutting endonucleases, called meganucleases, which recognize and cleave a specific nucleotide sequence, to polynucleotide sequences encoding for said rare-cutting endonucleases, to a vector comprising one of said polynucleotide sequences, to a cell or animal comprising one of said polynucleotide sequences or said rare-cutting endonucleases, to a process for producing one of said rare-cutting endonucleases and any use of the disclosed products and methods. More particularly, this invention contemplates any use of such rare-cutting endonuclease for genetic engineering and gene therapy. | 06-17-2010 |
20100146651 | MEGANUCLEASE VARIANTS CLEAVING A DNA TARGET SEQUENCE FROM THE HPRT GENE AND USES THEREOF - An I-CreI variant or a single-chain derivative having at least one substitution in one of the two functional subdomains of the LAGLIDADG (SEQ ID NO: 153) core domain situated from positions 26 to 40 and 44 to 77 of I-CreI, and being able to cleave a DNA target sequence from the HPRT gene having a nucleotide sequence of SEQ ID NO: 1 to 14. Use of said variant for inducing a site-specific modification in the HPRT gene, for therapeutic (gene therapy of Lesch-Nyhan syndrome) or non-therapeutic purpose (engineering of transgenic animals and recombinant cell lines). | 06-10-2010 |
20100144012 | USE OF MEGANUCLEASES FOR INDUCING HOMOLOGOUS RECOMBINATION EX VIVO AND IN TOTO IN VERTEBRATE SOMATIC TISSUES AND APPLICATION THEREOF - A single chain homing endonuclease, comprising a first variant of I-CreI having the amino acid sequence of accession number pdb 1g9y and a second variant of I-CreI variant having the amino acid sequence of accession number pdb 1g9y in a single polypeptide. | 06-10-2010 |
20100086533 | LAGLIDADG HOMING ENDONUCLEASE VARIANTS HAVING NOVEL SUBSTRATE SPECIFICITY AND USE THEREOF - A LAGLIDADG homing endonuclease variant having novel substrate specificity, said variant being obtainable by a method comprising: (a) the mutation of at least one amino acid residue of the final C-terminal loop of a parent LAGLIDADG homing endonuclease, with the exclusion of the threonine 140 of I-CreI, b) the selection and/or screening of the variants from step (a) having a pattern of cleaved DNA targets that is different from that of the parent LAGLIDADG homing endonuclease. | 04-08-2010 |
20100022550 | TETRAHYDROCARBAZOLE DERIVATIVES USEFUL AS ANDROGEN RECEPTOR MODULATORS - The present invention provides a compound of the formula: Formula (I) or a pharmaceutically acceptable salt thereof; pharmaceutical compositions comprising an effective amount of a compound of Formula (I) in combination with a suitable carrier, diluent, or excipient; and methods for treating physiological disorders, particularly frailty, osteoporosis, osteopenia, and male and female sexual dysfunction comprising administering to a patient in need thereof an effective amount of a compound of formula (I). | 01-28-2010 |
20100021448 | I-CREI MEGANUCLEASE VARIANTS WITH MODIFIED SPECIFICITY, METHOD OF PREPARATION AND USES THEREOF - Method of preparing 1-CreI meganuclease variants having a modified cleavage specificity, variants obtainable by said method and their applications either for cleaving new DNA target or for genetic engineering and genome engineering for non-therapeutic purposes. Nucleic acids encoding said variants, expression cassettes comprising said nucleic acids, vectors comprising said expression cassettes, cells or organisms, plants or animals except humans, transformed by said vectors. | 01-28-2010 |
20090271881 | MEGANUCLEASE VARIANTS CLEAVING A DNA TARGET SEQUENCE FROM A RAG GENE AND USES THEREOF - An I-CreI variant, wherein one of the I-CreI monomers has at least two substitutions, one in each of the two functional subdomains of the LAGLIDADG core domain situated respectively from positions 26 to 40 and 44 to 77 of I-CreI, said variant being able to cleave a DNA target sequence from a RAG gene. Use of said variant and derived products for the prevention and the treatment of a SCID syndrome associated with a mutation in a RAG gene. | 10-29-2009 |
20090222937 | MEGANUCLEASE VARIANTS CLEAVING A DNA TARGET SEQUENCE FROM A XERODERMA PIGMENTOSUM GENE AND USES THEREOF - An I-CreI variant which has at least two substitutions, one in each of the two functional subdomains of the LAGLIDADG (SEQ ID NO: 229) core domain situated from positions 26 to 40 and 44 to 77 of I-CreI, said variant being able to cleave a DNA target sequence from a xeroderma pigmentosum gene. Use of said variant and derived products for the prevention and the treatment of Xeroderma pigmentosum. | 09-03-2009 |
20090220476 | LAGLIDADG HOMING ENDONUCLEASE VARIANTS HAVING MUTATIONS IN TWO FUNCTIONAL SUBDOMAINS AND USE THEREOF - Provided is a LAGLIDADG (SEQ ID NO: 50) homing endonuclease variant having mutations in two separate subdomains, each binding to a distinct part of a modified DNA target half-site, the LAGLIDADG (SEQ ID NO: 50) homing endonuclease variant being able to cleave a chimeric DNA target sequence having nucleotides bound by each subdomain. A heterodimeric meganuclease and derived products for genetic engineering, genome therapy and antiviral therapy are also provided. | 09-03-2009 |
20080271166 | I-Dmoi Derivatives with Enhanced Activity at 37oC and Use Thereof - I-DmoI derivatives with enhanced cleavage activity at 37° C., said mutant comprising a sequence of a mutant of a I-DmoI endonuclease or a chimeric 5 derivative thereof including at least the first I-DmoIdomain, said sequence comprising the sub-situation of at least: (i) one of the residues in positions 4, 20, 49, 52, 92, 94 and/or 95 of said first I-DmoIdomain, and/or (ii) one of the residues in positions 101, 102, and/or 109 of the linker or the beginning of the second domain of I-DmoI, if present. 10 Polynucleotide encoding said derivatives, cell, animal or plant comprising said polynucleotide and use thereof for isolating meganucleases with new DNA target specificity. | 10-30-2008 |