Abstract: The invention describes a method for obtaining purified recombinant Adeno-Associated Virus particles (rAAV), comprising performing a depth filtration of a starting material previously obtained from cells producing rAAV particles comprising a cell lysate and/or a culture supernatant, followed by a first step of anion-exchange chromatography, a second step of anion-exchange chromatography and a final step of tangential flow filtration, whereby purified recombinant Adeno-Associated Virus particles (rAAV) are provided.

Abstract: The invention describes a method for obtaining purified recombinant Adeno-Associated Virus particles (rAAV), comprising the steps of: a) performing a depth filtration of a starting material previously obtained from cells producing rAAV particles, the said starting material being selected in a group comprising a cell lysate and a culture supernatant, whereby a rAAV-containing clarified composition is provided; b) submitting the rAAV-containing clarified composition to an affinity purification step, whereby a first rAAV enriched composition is provided; c) submitting the first rAAV enriched composition at least once to: c1) a step of anion-exchange chromatography on a chromatographic support wherein elution is performed by using a salt gradient, preferably a linear salt gradient, and wherein the rAAV-containing fraction is collected, whereby a second rAAV enriched composition is provided; or c2) a step of density gradient centrifugation, wherein the rAAV-containing fraction is collected, whereby a second rAAV e

Abstract: The present invention relates to a method for generating neuronal and muscular cells from pluripotent stem cells.

Abstract: A compound for use in the treatment and/or prevention of diseases or disorders wherein an inhibition of protein prenylation is required, which is alternatively chosen among wherein R1 is 2-pyridyl, 3-pyridyl or 4-pyridyl; R2 represents: and wherein R5 and R6 represent a hydrogen atom or a (C1-4)alkyl group; and R7 represents an arylcarbonyl group, a heteroarylcarbonyl group, a heteroarylacetyl group, a (C1-C4)alkoxy-carbonylmethyl group, a group. Also disclosed are novel compounds of formulae (I) and (II).

Abstract: A compound for use in the treatment and/or prevention of diseases or disorders wherein an inhibition of protein prenylation is required, which is alternatively chosen among wherein R1 is 2-pyridyl, 3-pyridyl or 4-pyridyl; R2 represents: and wherein R5 and R6 represent a hydrogen atom or a (C1-4)alkyl group; and R7 represents an arylcarbonyl group, a heteroarylcarbonyl group, a heteroarylacetyl group, a (C1-C4)alkoxy-carbonylmethyl group, a group. Also disclosed are novel compounds of formulae (I) and (II).

Abstract: The invention concerns decorin for increasing muscle mass, particularly in the treatment of muscular dystrophies.

Abstract: The invention relates to the use of fibromodulin and lumican, particularly active fragments thereof, to increase muscle mass, especially in the treatment of muscular dystrophies.

Abstract: The invention relates to the use of a hydroxymethylglutaryl-coenzyme A (HMG-CoA) reductase inhibitor and of a farnesyl-pyrophosphate synthase inhibitor, or of one of their associated physiologically acceptable salts, in the preparation of a composition, particularly a pharmaceutical composition, for use in the treatment of human or animal, pathological or nonpathological situations related to the accumulation and/or the persistence of prenylated proteins in cells, such as during progeria (Hutchinson-Gilford syndrome), restrictive dermopathy or physiological ageing.

Abstract: The present invention relates to an ex vivo method for preparing induced paraxial mesoderm progenitor (iPAM) cells, said method comprising the step of culturing pluripotent cells in an appropriate culture medium comprising an effective amount of an activator of the Wnt signaling pathway and an effective amount of an inhibitor of the Bone Morphogenetic Protein (BMP) signaling pathway.

Abstract: The invention relates to a method for reprogramming target cells to multipotent progenitor cells capable of differentiating into muscular, skeletal or dermal cell lines. In particular, the invention relates to an ex vivo method for preparing induced presomitic mesoderm (iPSM) cells, said method comprising the steps of: a) providing target cells to be reprogrammed, and, b) culturing said target cells under appropriate conditions for reprogramming said target cells into iPSM cells, wherein said appropriate conditions comprises increasing expression of at least one T-Box transcription factor in said target cells. The invention further relates to the use of said iPSM cells, for example, for regenerating skeletal, muscle, dermal and cartilage tissues.

Abstract: The invention concerns decorin for increasing muscle mass, particularly in the treatment of muscular dystrophies.

Abstract: The invention concerns decorin for increasing muscle mass, particularly in the treatment of muscular dystrophies.

Abstract: The invention relates to a culture medium comprising an inhibitor of the BMP signaling pathway; and an inhibitor of the TGF/activin/nodal signaling pathway and to a method for obtaining a population of neural precursors using said culture medium.

Abstract: A method for obtaining cell populations derived from the muscular tissue and their use for preparing cell therapy products includes culturing cells previously removed by biopsy from skeletal muscular tissues, identifying the different types of cells present at different stages of culture, selecting the culture stage on the basis of the required cell population and collecting the selected culture stage for preparing a cell therapy product. The invention also concerns cell populations derived from muscular tissue obtained by implementing the method whereof the dominant cell type is CD34+, CD15+ or CD56+ or Class 1+HLA, or comprises a doubly negative CD56?/CD15? cell type or may comprise more minority CD10+, Stro-1+ and CD 117+ cell types.

Abstract: The invention relates to the technical fields of biology, pharmacology and medicine. The invention is suitable for use in particular in the human and animal health fields. More specifically, the invention relates to the use of a cAMP modulator in the preparation of compositions that are intended for the prevention or treatment of peripheral neuropathies. The invention further relates to tools and kits to prepare the aforementioned compositions.

Abstract: A method for obtaining cell populations derived from the muscular tissue and their use for preparing cell therapy products includes culturing cells previously removed by biopsy from skeletal muscular tissues, identifying the different types of cells present at different stages of culture, selecting the culture stage on the basis of the required cell population and collecting the selected culture stage for preparing a cell therapy product. The invention also concerns cell populations derived from muscular tissue obtained by implementing the method whereof the dominant cell type is CD34+, CD15+ or CD56+ or Class 1+ HLA, or comprises a doubly negative CD56?/CD15? cell type or may comprise more minority CD10+, Stro-1+ and CD117+ cell types.

Abstract: Described is the use of magnesium (Mg2+) for the preparation of a therapeutic composition for the introduction of a polynucleotide into a cell in vivo.

Abstract: A nucleic acid sequence comprising: 1) the sequence represented in FIG. 8; or 2) the sequence represented in FIG. 2; or 3) a part of the sequence of FIG. 2 with the proviso that it is able to code for a protein having a calcium dependant protease activity involved in a LGMD2; or 4) a sequence derived from a sequence defined in 1), 2) or 3) by substitution, deletion or addition of one or more nucleotides with the proviso that said sequences still codes for said protease.

Abstract: Described is the use of a nuclease inhibitor or of interleukin-10 (IL-10) for the preparation of a therapeutic composition for improving transfection of a polynucleotide into a cell, and to compositions comprising a mixture of polynucleotide and nuclease inhibitor and/or interleukin-10.

Abstract: A method for detecting the homozygous or heterozygous state of mutations assumed to be present in a nucleic acid by simultaneously using two primer pairs. The two different primer pairs lead to the production of amplified fragments of different sizes, and the number and quality of the amplified bands enables homozygous and heterozygous items to be distinguished on the basis of said mutation.