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 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 present invention relates to pharmaceutical compositions that act on the p53 protein or its gene and methods of use for the treatment of neurodegenerative diseases.

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 present invention relates to nucleic acid sequences producing at least one functional miRNA, at least one functional shRNA and/or at least one functional siRNA, said miRNA, shRNA or siRNA being designed to silence the expression of a gene that encodes a cholinergic protein. The present invention further relates to compositions and kits comprising such nucleic acid sequences as well as to uses thereof.

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 present invention relates to nucleic acid sequences producing at least one functional miRNA, at least one functional shRNA and/or at least one functional siRNA, said miRNA, shRNA or siRNA being designed to silence the expression of a gene that encodes a cholinergic protein. The present invention further relates to compositions and kits comprising such nucleic acid sequences as well as to uses thereof.

Abstract: The present invention relates to method for preventing, treating or alleviating a central nervous system (CNS) disorder using a non replicative lentivirus comprising a lentiviral genome comprising a nucleic acid sequence producing at least one functional miRNA, at least one functional shRNA and/or at least one functional siRNA, preferably derived from said shRNA, said miRNA, shRNA or siRNA being designed to silence the expression of a gene that encodes a protein of the astrocyte cytoskeleton. The present invention further relates to compositions and kits comprising such a lentivirus as well as to uses thereof.

Abstract: The present invention relates to a tetracycline dependent gene regulatory system or composition controlling the expression of a target gene in a cell and to methods using said system or composition. The present invention more specifically discloses compositions, vectors and methods allowing tetracycline-controlled expression of short-hairpin RNAs (shRNAs), and demonstrates inducible, reversible and stable RNA interference (RNAi) using the same in a cell. The invention can be used to cause reversible control of the expression of any gene and may therefore find applications in the fields of mammalian, in particular human, genetics and molecular therapeutics, in cell and gene therapy, research as well as in genetic studies using transgenic animals.

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 present invention is related to compositions and methods for the delivery of nucleic acids to neurons in a mammal, and uses thereof. The present invention specifically discloses the use of compounds that cause synaptic nerve sprouting to increase neuron retrograde transport of a vector or a product (a polypeptide or a nucleic acid for example) in a mammal. The invention is also based on the use of a compound that interacts with synaptosomal associated proteins to increase neuron retrograde transport of a vector or a product such as one cited above in a mammal. The invention also relates to a product comprising a viral vector comprising a transgene and a compound that causes synaptic nerve sprouting, for sequential use for delivering said transgene to neurons by retrograde transport and its uses for the preparation of a composition used as a treatment in several neurological disorders.

Abstract: The present invention relates to pharmaceutical compositions that act on the p53 protein or its gene and methods of use for the treatment of neurodegenerative diseases.

Abstract: The present invention relates to pharmaceutical compositions that act on the p53 protein or its gene and methods of use for the treatment of neurodegenerative diseases.

Abstract: The present invention relates to a defective adenovirus comprising at least a DNA sequence coding for all or an active part of glutathione peroxidase or a derivative thereof. It also relates to their utilization in therapy and to the corresponding pharmaceutical compositions.

Abstract: The invention concerns novel methods and constructs for controlling nucleic acid expression, in particular methods and constructs using NRSE sequences for obtaining a targeted expression of transgenes in nerve cells.

Abstract: The invention concerns novel recombinant viruses and their use for gene-transfer into nerve cells of vertebrates. The invention also concerns pharmaceutical compositions comprising said recombinant viruses. More particularly, the invention concerns novel vectors derived from baculoviruses and their use for treating diseases of the nervous system of vertebrates.

Abstract: The present invention relates to vectors, compositions and methods for delivering transgenes into mammalian cells. The invention also relates to genetic constructs and recombinant cells suitable to produce such transgenes. The invention more particularly relates to a vector suitable for transgene delivery into mammalian cells, wherein said vector comprises a chimeric genetic construct comprising a transgene operably linked to at least two distinct posttranscriptional regulatory elements functional in mammalian cells. This invention can be used in experimental, research, therapeutic, prophylactic or diagnostic areas.

Abstract: The present invention relates to nucleic acids that encode novel basic helix-loop-helix polypeptides. The present invention also relates to novel basic helix-loop-helix polypeptides, in particular, rat Relax and human ngn3 polypeptides. The invention also relates to methods for the detection of nucleic acids and polypeptides according to the invention. The invention also relates to methods for the detection of activators or inhibitors of the polypeptides of the invention. Finally, the present invention relates to methods of prevention and/or treatment of pathologies, dysfunctions, disorders or conditions affecting the nervous system.

Abstract: A defective recombinant adenovirus including at least one DNA sequence coding for all or an active part of a superoxide dismutase or a derivative thereof. The therapeutical use thereof and corresponding pharmaceutical compositions are also disclosed.