Abstract: The present invention relates to at least one glycosylation inhibitor for use in combination with CAR cell therapy. Preferably the glycosylation inhibitor improves the therapeutic potential of the CAR cell therapy. The invention also relates to pharmaceutical composition and to population or subpopulation of CAR cell that has been contacted with at least one glycosylation inhibitor.

Abstract: The present invention relates to an antibody or antigen binding fragment thereof that binds specifically to IGFBP3 and does not displace the binding of IGF-I to IGFBP3. The antibody inhibits or reduces the binding of IGFBP3 to the TMEM219 receptor. The invention also relates to methods for their production, pharmaceutical compositions containing said antibodies, and uses thereof.

Abstract: The present invention relates to a method for early detection of artificial pump dysfunction based on a time-frequency analysis of the pump motor power consumption (PRC). The method allows to prevent low output syndrome, cardiogenic shock, pump thrombus and/or cardiac arrest or death as well as to monitor the efficacy of a thrombolytic therapy and/or to optimize intensity and duration of a thrombolytic therapy.

Abstract: The present invention relates to an isolated polynucleotide comprising from 5? to 3?: a first homology region, a nucleotide sequence encoding a RAG1 polypeptide or a RAG1 polypeptide fragment, and a second homology region for use in treating a RAG-deficient immunodeficiency.

Abstract: Viral vector production processes and methods of purifying a viral vector from a host cell are provided herein.

Abstract: A product comprising two or more artificial transcription repressors (ATRs), or polynucleotides encoding therefor, selected from groups (a), (b), (c) or (d): (a) an ATR comprising a DNA-binding domain operably linked to a KRAB domain or homologue thereof; (b) an ATR comprising a DNA-binding domain operably linked to a DNMT3A, DNMT3B or DNMT1 domain or homologue thereof; (c) an ATR comprising a DNA-binding domain operably linked to a DNMT3L domain or homologue thereof; and (d) an ATR comprising a DNA-binding domain operably linked to a SETDB1 domain or homologue thereof, wherein at least two of the ATRs are selected from different groups (a), (b), (c) or (d).

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 inhibitor of p53 activation for use in haematopoietic stem and/or progenitor cell gene therapy, preferably wherein the inhibitor is an inhibitor of p53 phosphorylation, more preferably an inhibitor of p53 Serine 15 phosphorylation.

Abstract: An image capturing method (100) using an image capturing apparatus (1) that comprises an imaging device (2) having an image catcher (3) for generating image data, the method (100) comprising displaying (S101), using an image reproducing device (4), images based on the image data, generating (S102) attribute data from a plurality of biological signals measured by a plurality of biological sensors (6), the attribute data being at least an information extracted by one or more biological signals through a computational processing and being indicative of attributes of a user at a time that the user is viewing the images displayed by the image reproducing device (4), generating (S103) significance data based on the attribute data, and associating the attribute data from which the significance data is generated with each displayed image, wherein the significance data are generated by comparing the attribute data with predetermined significance threshold values in order to identify a significance state for each attri

Abstract: The invention features compositions and methods for the treatment and prevention of peroxisomal diseases (e.g., neonatal adrenoleukodystrophy and Zellweger syndrome), including in a subject selected as having increased levels of metallothionein polypeptides. The invention also provides compositions and methods for identifying a subject having a peroxisomal disease involving detecting metallothionein polypeptides or polynucleotides.

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: 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: 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.