Abstract: The present invention provides compositions and methods for the treatment or prevention of a lysosomal disease or disorder involving increasing the level, expression, or activity of a metallothionein polypeptide or polynucleotide in the subject.

Abstract: The invention is based upon the discovery that T regulatory type 1 (Tr1) cells express particular cell surface markers that allow for their selection, enrichment, isolation, purification and administration. The ability to use the particular markers described herein to select, enrich, isolate, purify and administer Tr1 cells allows for improved methods of Tr1 therapies for treating a wide variety of diseases and disorders.

Abstract: A gene vector for use in gene therapy comprising at least one miRNA sequence target operably linked to a nucleotide sequence having a corresponding miRNA in a hematopoietic progenitor cell (HSPC) or hematopoietic stem cell (HSC) which prevents or reduces expression of the nucleotide sequence in a HSPC or HSC but not in a differentiated cell.

Abstract: The invention is based upon the discovery that T regulatory type 1 (Tr1) cells express particular cell surface markers that allow for their selection, enrichment, isolation, purification and administration. The ability to use the particular markers described herein to select, enrich, isolate, purify and administer Tr1 cells allows for improved methods of Tr1 therapies for treating a wide variety of diseases and disorders.

Abstract: Type 1 diabetes mellitus is characterized by loss of pancreatic insulin-producing beta cells, resulting in insulin deficiency. The usual cause of this beta cell loss is autoimmune destruction. The inventors provide the first evidence of a causal link between influenza virus infection and the development of type 1 diabetes and/or pancreatitis. This causal link between infection and type 1 diabetes and/or pancreatitis provides various therapeutic, prophylactic and diagnostic opportunities.

Abstract: A gene vector comprising a miRNA sequence target.

Abstract: An effective Pseudomonas aeruginosa vaccine may require one or several antigenic components, and so various antigens of P. aeruginosa are identified for use in immunization. These polypeptides may optionally be used in combination with other nosocomial antigens.

Abstract: It is described in vitro methods for expanding, detecting or isolating rare populations of antigen specific memory T cells. It is also described an in vitro method for obtaining a genetically modified memory T cell population. Uses of cells so obtained are also disclosed.

Abstract: A gene vector for use in gene therapy comprising at least one miRNA sequence target operably linked to a nucleotide sequence having a corresponding miRNA in a hematopoietic progenitor cell (HSPC) or hematopoietic stem cell (HSC) which prevents or reduces expression of the nucleotide sequence in a HSPC or HSC but not in a differentiated cell.

Abstract: The present invention relates to a tolerogenic dendritic cell population (Tr-DC) capable of generating a population of T cells having regulatory activity, method of production and uses thereof. Furthermore, soluble HLA-G promotes the differentiation of a population of T cells with regulatory activity.

Abstract: A gene vector adapted for transient expression of a transgene in a peripheral organ cell comprising a regulatory sequence operably linked to a transgene wherein the regulatory sequence prevents or reduces expression of said transgene in hematopoietic lineage cells.

Abstract: A gene vector for use in gene therapy comprising at least one miRNA sequence target operably linked to a nucleotide sequence having a corresponding miRNA in a hematopoietic progenitor cell (HSPC) or hematopoietic stem cell (HSC) which prevents or reduces expression of the nucleotide sequence in a HSPC or HSC but not in a differentiated cell.

Abstract: The instant invention refers to an optical method to extrapolate cell membrane conductance by indirect measurement of changes in transmembrane voltage, upon exposure of a cell sample to electric current pulses. The method is advantageously used for evaluating the activity of molecules able to alter, directly or indirectly, membrane permeability. A specific field of application is the screening of candidate compounds putatively acting on ion channel activity. In particular, it is open to the study of all ion channels with no limitations on the mechanisms of activation or to the ion species involved. The method is also advantageously used for evaluating a cell status, namely a differentiative or a pathologic status.

Abstract: The present invention relates to a tolerogenic dendritic cell population (Tr-DC) capable of generating a population of T cells having regulatory activity, method of production and uses thereof. Furthermore, soluble HLA-G promotes the differentiation of a population of T cells with regulatory activity.

Abstract: Type 1 diabetes mellitus is characterized by loss of pancreatic insulin-producing beta cells, resulting in insulin deficiency. The usual cause of this beta cell loss is autoimmune destruction. The inventors provide the first evidence of a causal link between influenza virus infection and the development of type 1 diabetes and/or pancreatitis. This causal link between infection and type 1 diabetes and/or pancreatitis provides various therapeutic, prophylactic and diagnostic opportunities.

Abstract: It is described in vitro methods for expanding, detecting or isolating rare populations of antigen specific memory T cells. It is also described an in vitro method for obtaining a genetically modified memory T cell population. Uses of cells so obtained are also disclosed.

Abstract: The present invention relates to a method for the diagnostic and/or prognostic of a neurological disease characterized by an inflammation process in a subject comprising measuring the amount of myeloid derived microvesicles in a cerebrospinal fluid sample obtained from the subject. The invention further relates to a method for predicting and/or monitoring the efficacy of a treatment for a neurological pathology or for monitoring a neurological disease progression.

Abstract: Disclosed herein are methods and compositions for inactivating TCR genes, using zinc finger nucleases (ZFNs) comprising a zinc finger protein and a cleavage domain or cleavage half-domain in conditions able to preserve cell viability. Polynucleotides encoding ZFNs, vectors comprising polynucleotides encoding ZFNs and cells comprising polynucleotides encoding ZFNs and/or cells comprising ZFNs are also provided. Disclosed herein are also methods and compositions for expressing a functional exogenous TCR in the absence of endogenous TCR expression in T lymphocytes, including lymphocytes with a central memory phenotype. Polynucleotides encoding exogenous TCR, vectors comprising polynucleotides encoding exogenous TCR and cells comprising polynucleotides encoding exogenous TCR and/or cells comprising exogenous TCR are also provided.

Abstract: Disclosed herein are methods and compositions for targeted integration of one or more copies of a sequence of interest using zinc finger nucleases (ZFNs) comprising a zinc finger protein and a cleavage domain or cleavage half-domain and integrase defective lentiviral donor constructs.

Abstract: The present invention relates to a method for reprogramming a differentiated non neuronal cell into a dopaminergic neuron comprising the step of inducing the expression in the differentiated non neuronal cell of at least the protein encoded by the Mash1 human gene or orthologues thereof and the protein encoded by the Nurr1 human gene or orthologues thereof, expression vectors, reprogrammed dopaminergic neuron and uses thereof.