Abstract: The present invention provides compositions and methods for the treatment or prevention of a neurological disease or disorder of the central nervous system (e.g., a storage disorder, lysosomal storage disorder, neurodegenerative disease, etc.) by reconstitution of brain myeloid cell and microglia upon transplantation of hematopoietic cells enriched in microglia reconstitution potential. The invention also provides compositions and methods for ablating and reconstituting microglia.

Abstract: An enveloped viral particle producer or packaging cell, wherein the cell is genetically engineered to decrease expression of MHC-I on the surface of the cell.

Abstract: A cartridge is adapted to house at least one biological sample therein, where the cartridge contains at least two overlapping layers, and the layers include at least one layer of highly hydrophobic, inert, and biocompatible material, with contact angle ?c?90°, hydrophobic layer, and optionally, at least one layer of double-sided adhesive material, where in the absence of the at least one layer of double-sided adhesive material, the overlapping layers are connected together by chemical and/or physical bonding, where each of the overlapping layers has at least one inner hole that is pervious when the layers overlap one another, and the at least one inner hole is closed by the at least one biological sample, where loaded in the cartridge.

Abstract: A gene vector comprising a miRNA sequence target.

Abstract: A stand-alone continuous positive airways pressure, CPAP, apparatus having a face-mask and a connected electro-mechanical device to supply air to the face-mask is disclosed. The electro-mechanical device includes a pneumatic channel for flowing air to be delivered to the face mask and a control unit for managing the air pressure of the air inside the pneumatic channel. The CPAP apparatus includes a turbine fan, located in the electro-mechanical device housing, connected to the control unit for pressurizing atmospheric air. The pneumatic channel includes an inlet portion located upstream of the turbine fan to receive atmospheric air, and an outlet portion located downstream of the turbine fan to deliver the pressurized air to the face-mask through an outlet opening. The pneumatic channel also longitudinally extends from the inlet portion to the outlet portion.

Abstract: The present invention relates to a conjugate comprising a first peptide of sequence CNGRCG (SEQ ID NO: 1) linked to the N-terminus of a protein and a compound X linked to the N-terminus of said peptide and to related medical uses.

Abstract: The present invention relates to an inhibitor of asparagine synthase for use for the treatment of a disorder characterized by renal and/or liver cyst formation and relative pharmaceutical composition.

Abstract: The present invention refers to a method to produce a T cell with advantageous properties. The invention also refers to a T cell or an engineered T cell produced by the method and its use in therapy.

Abstract: The present invention relates to compounds that are inhibitors of hepatitis B virus (HBV). Compounds of this invention are useful alone or in combination with other agents for treating, ameliorating, preventing or curing HBV infection and related conditions. The present invention also relates to pharmaceutical compositions containing the compounds.

Abstract: The present invention refers to an inhibitor of substance P and/or of its receptor for use in the treatment and/or prevention of stem cell deficiency. Preferably the inhibitor is an NK1 antagonist. Preferably the stem cell deficiency is a corneal epithelial stem cell deficiency. The invention also refers to pharmaceutical compositions containing the inhibitor for use in the treatment and/or prevention of stem cell deficiency.

Abstract: TCR AND PEPTIDES A T-cell receptor (TCR), which binds to a Wilms tumour 1 protein (WT1) peptide when presented by a major histocompatibility complex (MHC).

Abstract: The present invention relates to methods for the screening, diagnosis and/or prognosis of renal cell carcinoma. The methods of the invention are based on the determination of expression profiles of sets of miRNAs representative for renal cell carcinoma optionally associated with genotype analysis, in particular of uromodulin SNP variants. The invention also refers to a kit to perform such methods.

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 present invention refers to chromogranin A-derived peptides that are potent dual ligands for integrins ?v?6 and av?8, their therapeutic and diagnostic uses and relative compositions.

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 compounds that are inhibitors of hepatitis B virus (HBV). Compounds of this invention are useful alone or in combination with other agents for treating, ameliorating, preventing or curing HBV infection and related conditions. The present invention also relates to pharmaceutical compositions containing the compounds.

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: Use of (i) an anti-CD3 antibody, (ii) an anti-CD56 antibody, (iii) an anti-CD14 antibody, (iv) an anti-CD38 antibody, (v) an anti-CD45 antibody, (vi) an anti-CD90 antibody, (vii) an anti-CD135 antibody, (viii) an anti-CD10 antibody, (ix) an anti-CD11c antibody, (x) an anti-CD19 antibody, (xi) an anti-CD34 antibody, (xii) an anti-CD45RA antibody, (xiii) an anti-CD7 antibody, (xiv) an anti-CD71 antibody, (xv) an anti-CD41/CD61 complex antibody or an anti-CD41 antibody and/or an anti-CD61 antibody (xvi) an anti-CD33 antibody and/or an anti-CD66b antibody, for identifying hematopoietic cell subtypes in an isolated sample, determining the relative frequency of hematopoietic cell subtypes in an isolated sample and/or quantifying the number of cells within hematopoietic cell subtypes in an isolated sample, wherein each of (i) to (xvi) is labelled with a different fluorochrome, wherein when (xvi) is an anti-CD33 antibody and an anti-CD66b antibody, the anti-CD33 antibody and anti-CD66b are labelled with the same fluo

Abstract: The present invention relates to compounds that are inhibitors of hepatitis B virus (HBV). Compounds of this invention are useful alone or in combination with other agents for treating, ameliorating, preventing or curing HBV infection and related conditions. The present invention also relates to pharmaceutical compositions containing the compounds.

Abstract: The present invention relates to an inhibitor of DUX4 and its use, in particular in the prevention and/or treatment of a condition associated with an aberrant expression and/or function of at least one DUX4 protein and/or of at least one DUX4 fusion protein. Preferably the inhibitor is MATRIN-3 (MATR3), fragment, variant, fusion, or conjugate thereof. The invention also relates to a pharmaceutical composition comprising such inhibitor, to vector and nucleic acids.