Abstract: The present invention is in the field of the gene editing molecular tools. The present invention relates to rewritten nucleic acid sequences encoding repeated DNA recognition motifs of TALE (Transcription Activator-Like Effector) proteins. These nucleic acid sequences allow assembly and cloning of TALE repeats in any type of vectors, especially viral vectors. The invention thereby contributes to improving gene targeting in cells using TALE derived proteins, in particular for genetic regulation or modification. The present invention is particularly drawn to virus mediated transformation methods, by providing vectors, compositions and kits including said new nucleic acid sequences.

Abstract: 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.

Abstract: Use of meganucleases for cleaving DNA in a non-human or an isolated human cell and, in some instances, inducing homologous recombination in said cells and to its application for genome engineering and gene therapy.

Abstract: A monomer of an I-CreI meganuclease variant wherein said monomer comprises mutations in the amino acid sequence of SEQ ID NO: 34, wherein said mutations include, (i) at least one and up to five amino acid substitutions from residue Q44 to residue R70, said substitutions selected from the group consisting of substitutions at positions Q44, T46, Y66, R68 and R7; and (ii) at least one and up to six amino acid substitutions from residue Q26 to residue Q38 said substitutions selected from the group consisting of substitutions at positions Q26, K28, N30, S32, Y33 and Q38, and wherein said monomer when in dimeric form binds and cleaves a DNA target sequence. Said dimeric forms include homodimeric, heterodimeric and single-chain I-CreI meganuclease variants.

Abstract: The present invention relates to a polynucleotide encoding a monomer of an I-CreI variant said monomer comprising mutations in the amino acid sequence of SEQ ID NO: 34, wherein said mutations include: (i) at least one and up to eleven amino acid substitutions from residue S22 to Q44 said substitutions selected from the group consisting of substitutions at positions I24, Q26, K28, N30, S32, Y33, Q38, S40 and T42; and (ii) at least one and up to six amino acid substitutions from residue Y66 to I77 said substitutions selected from the group consisting of substitutions at positions Y66, R68, R70, V73 and I77; and wherein said monomer when in dimeric form binds and cleaves DNA.

Abstract: 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.

Abstract: 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.

Abstract: 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.

Abstract: A monomer of an I-CreI meganuclease variant wherein said monomer when in dimeric form binds and cleaves DNA.

Abstract: A monomer of an I-CreI meganuclease variant wherein said monomer when in dimeric form binds and cleaves DNA.

Abstract: A monomer of an I-CreI meganuclease variant wherein said monomer when in dimeric form binds and cleaves DNA.

Abstract: Use of meganucleases for inducing homologous recombination ex vivo and in toto in vertebrate somatic tissues and to its application for genome engineering and gene therapy.

Abstract: Use of meganucleases for inducing homologous recombination ex vivo and in toto in vertebrate somatic tissues and to its application for genome engineering and gene therapy.

Abstract: Use of meganucleases for inducing homologous recombination ex vivo and in toto in vertebrate somatic tissues and to its application for genome engineering and gene therapy.

Abstract: 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.

Abstract: 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.

Abstract: 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.

Abstract: 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.

Abstract: A single chain homing endonuclease, comprising a first variant of I-CreI having the amino acid sequence of accession number pdb 1g9y (SEQ ID NO: 23 is residues 1-153 of pdb Ig9y) and a second variant of I-CreI variant having the amino acid sequence of accession number pdb 1g9y (SEQ ID NO: 23 is residues 1-153 of pdb Ig9y) in a single polypeptide.

Abstract: 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.