Abstract: The present invention relates to a nucleic acid for the controlled expression of a nucleic acid encoding a Cas nuclease in an individual, comprising (i) a regulatory polynucleotide comprising a minimal promoter and from one to twenty AARE nucleic acids, and (ii) a nucleic acid encoding a Cas nuclease, which is placed under the control of the regulatory polynucleotide,

Abstract: The present invention relates to genome editing by the mean of Cas nucleases. The inventors found that the expression of Cas nucleases may be finely controlled by the use of regulatory elements comprising a minimal promoter and at least one amino acid response element (AARE) nucleic acid, which are responsive to a diet deficient in at least one essential amino acid, or tunicamycin. For example, a FLAG-Cas9-GFP fusion and a Cas9-FLAG-RFP fusion could be expressed in 293 T cells. In addition, in the presence of a donor plasmid bearing a puromycin resistant gene, integration of the said puromycin resistant gene may be performed at the site of the safe harbour locus AASV1 on the genome of 293 T cells.

Abstract: The present invention relates to a lentiviral vector wherein the expressed integrase protein comprises at least one point mutation consisting of the substitution of the aspartic acid residue at position 167 by an amino acid selected from the group consisting of histidine, arginine and lysine.

Abstract: The present invention relates to at least one protein belonging to the bHLH family and/or at least a nucleic acid molecule coding for the bHLH proteins as drug.

Abstract: The invention concerns a non-integrative and non-replicative recombinant lentivirus as well as its uses, in particular for preparing a composition for transferring genes in vitro, ex vivo or in vivo. The invention is useful for transferring genes in any mammal organism, for example in liver, muscle, pancreas or central nervous system (including the ocular sphere) tissues or cells, and in particular for treating disorders or pathologies such as, for example, central nervous system, including the ocular sphere, disorders.

Abstract: The invention relates to a defective lentivirus which is pseudotyped with a lyssavirus envelope of the PV (rabies virus) or MOK type (Mokola virus), for example, and to the use thereof, especially in the preparation of a composition for in vivo transfer of genes in astrocytes and also for the treatment of disorders of the central nervous system.

Abstract: The invention concerns a non-integrative and non-replicative recombinant lentivirus as well as its uses, in particular for preparing a composition for transferring genes in vitro, ex vivo or in vivo. The invention is useful for transferring genes in any mammal organism, for example in liver, muscle, pancreas or central nervous system (including the ocular sphere) tissues or cells, and in particular for treating disorders or pathologies such as, for example, central nervous system, including the ocular sphere, disorders.

Abstract: The invention relates to a defective lentivirus which is pseudotyped with a lyssavirus envelope of the PV (rabies virus) or MOK type (Mokola virus), for example, and to the use thereof, especially in the preparation of a composition for in vivo transfer of genes in astrocytes and also for the treatment of disorders of the central nervous system.