Abstract: The present disclosure provides variant AAV8 capsids that exhibit altered capsid properties, e.g., improved transduction efficiency and/or specificity for the liver. The present disclosure further provides nucleic acids encoding the variant AAV8 capsids, recombinant AAV (rAAV) vectors comprising the variant AAV8 capsids, as well as host cells and compositions comprising the same. The present disclosure further provides methods of delivering a gene product to a subject and methods of treatment of a liver-borne blood disorder, the methods generally involving administering an effective amount of the rAAV vectors to a subject in need thereof.

Abstract: In order to prevent a rapidly rotating blade from flying away in the event of unintentional loosening, for which purpose the blade usually rotates in a protective blade housing, a catch pivot can-alternatively or additionally—be provided, which ends with its free end at such a small distance in front of an oppositely directed free end of a rotor and also extends in a direction of a blade axis of the blade 3 that a distance therebetween is less than a thickness of the blade. This prevents the blade from coming out radially through the distance between these two parts in the area of a central opening.

Abstract: An apparatus for generating a virtual image including a display element for generating an image, an optical waveguide for expanding an exit pupil, and an antiglare element, which is arranged after the optical waveguide in the beam path and is configured as a shutter comprising slats, wherein the slats are braced in pre-tensioning equipment outside of a visible region of the apparatus, and rest against a contacting element under pretension.

Abstract: The present invention relates to a polynucleotide comprising a Nuclear factor of activated T-cells (NFAT) binding site sequence and a reverse complement of said NFAT binding site sequence separated by a spacer sequence, to said polynucleotide for use in treating and/or preventing disease, and to viral particles, compositions, and uses related thereto. The present invention further relates to a polynucleotide comprising a Nuclear factor of activated T-cells (NFAT) binding site sequence and a reverse complement of said NFAT binding site sequence for use in treating and/or preventing an NFAT-mediated disease.

Abstract: The invention provides gene therapy vectors, such as adeno-associated virus (AAV) vectors, expressing a functional fragment of the miniaturized human micro-dystrophin gene and method of using these vectors to express the fragment of micro-dystrophin in skeletal muscles including diaphragm and cardiac muscle and to protect muscle fibers from injury, increase muscle strength and reduce and/or prevent fibrosis in subjects suffering from muscular dystrophy.

Abstract: The present invention relates to Abhydrolase containing domain 5 (ABHD5) and N-terminal fragments of HDAC4 (HDAC4-NT) and variants of the aforementioned peptides for the treatment and prevention of heart failure. The present invention further provides vectors for the cardiomyocyte-specific expression of said peptides and a test system comprising ABHD5 for the identification of novel compounds which are useful for the treatment of heart failure.

Abstract: The invention provides gene therapy vectors, such as adeno-associated vims (AAV) vectors, expressing a functional fragment of the miniaturized human micro-dystrophin gene and method of using these vectors to express the fragment of micro-dystrophin in skeletal muscles including diaphragm and cardiac muscle and to protect muscle fibers from injury, increase muscle strength and reduce and/or prevent fibrosis in subjects suffering from muscular dystrophy.

Abstract: The present invention relates to Abhydrolase containing domain 5 (ABHD5) and N-terminal fragments of HDAC4 (HDAC4-NT) and variants of the aforementioned peptides for the treatment and prevention of heart failure. The present invention further provides vectors for the cardiomyocyte-specific expression of said peptides and a test system comprising ABHD5 for the identification of novel compounds which are useful for the treatment of heart failure.

Abstract: The present invention relates to a compound decreasing the concentration of 2-hydroxy-glutarate (2HG) in a subject for use in treating, preventing, and/or preventing progression of cardiac remodeling, in particular cardiomyopathy and/or heart failure and to viral particles, compositions, uses and methods related thereto.

Abstract: A method for manufacturing a physical security element with a spatially appearing pattern includes a carrier and at least one see-through transparent cover layer. Onto the carrier is applied a design layer deformable under pressure. Either the carrier has a lower dimensional stability under heat than the cover layer, or a cover layer has a lower dimensional stability under heat than the carrier. A see-through transparent structural layer forming the pattern is arranged between carrier and cover layer. The structural layer has a higher dimensional stability under heat than either the carrier or a cover layer. During lamination, the structural layer is pressed into the carrier or into the cover layer, whereby the design layer is deformed under the pattern formed by the structural layer and the structural layer in its edge regions is reshaped in such a way that its surfaces, in cross-section, converge tangentially.

Abstract: The invention relates to an assembly module (20) for a motor vehicle (1), comprising an optical sensor system (30) which is suitable a) for monitoring a detection area (21) located outside of the vehicle (1), b) if a user (10) is detected in the detection area (21), for triggering a signal to commence an authentication check between an ID transponder (13) and the motor vehicle (1), c) for monitoring an actuation area (22) which is located outside of the vehicle (1) and which is different from the detection area (21), d) if a user (10) is detected in the actuation area (22), for providing a working signal for the vehicle (1), wherein an emergency actuation means (90) is provided for additionally triggering the working signal, which emergency actuation means is suitable e) for providing a working signal for the vehicle (1) via an activation of the emergency actuation means (90) by a body part (11, 12) of the user (10).

Abstract: The present invention relates to a modified viral structural protein (VSP) as a tool for specifically targeting a chimeric antigen receptor (CAR) expressed on cells of the immune system. The modified VSPs can assemble into virus like particles (VLP). Exposed areas of the VSPs are modified to comprise in a region located atthe surface of a higher order structure, e.g. such as a capsomeric structure, a capsid, a VLP, a viral vector or a virus, a ligand specifically binding to a CAR (LCAR). The present invention thus, provides a modified VSP. The invention also relates to a nucleic acid encoding said VSP. Further, the invention relates to a capsomeric structure, a capsid, a VLP, a viral vector or a virus comprising at least one VSP. Further, the invention relates to a pharmaceutical composition comprising the VSP, the nucleic acid, the capsomeric structure, the capsid, the VLP, the viral vector or the virus comprising at least one VSP.

Abstract: The present invention relates to a polynucleotide comprising a Nuclear factor of activated T-cells (NFAT) binding site sequence and a reverse complement of said NFAT binding site sequence separated by a spacer sequence, to said polynucleotide for use in treating and/or preventing disease, and to viral particles, compositions, and uses related thereto. The present invention further relates to a polynucleotide comprising a Nuclear factor of activated T-cells (NFAT) binding site sequence and a reverse complement of said NFAT binding site sequence for use in treating and/or preventing an NFAT-mediated disease.

Abstract: The present invention relates to an antagonist of the Two-Pore Domain Potassium Channel (TASK-1) K2P3.1 for use in the prevention and/or treatment of cardiac arrhythmia in a subject. The invention also relates to a nucleic acid molecule usable in the prevention and/or treatment of cardiac arrhythmia in a subject. The invention further relates to a cell comprising said nucleic acid molecule. The invention further relates to a vector comprising said nucleic acid molecule.

Abstract: A method for evaluating measurement data from a measurement of a plurality of workpieces includes obtaining a set of measurement data. Each workpiece has an associated set of measurement data. The set of measurement data corresponds to measurement points of the workpieces. The set of measurement data has, for each measurement point of the workpieces, at least one measured coordinate and/or, for each measured coordinate, a divergence from a comparison coordinate. The method includes determining a measure of the correlation of the measured coordinates and/or of the divergences is determined for a plurality of the sets of measurement data, in each case in relation to a pair of measurement points that consists of two measurement points of the workpieces.

Abstract: A method for manufacturing a physical security element with a spatially appearing pattern includes a carrier and at least one see-through transparent cover layer. Onto the carrier is applied a design layer deformable under pressure. Either the carrier has a lower dimensional stability under heat than the cover layer, or a cover layer has a lower dimensional stability under heat than the carrier. A see-through transparent structural layer forming the pattern is arranged between carrier and cover layer. The structural layer has a higher dimensional stability under heat than either the carrier or a cover layer. During lamination, the structural layer is pressed into the carrier or into the cover layer, whereby the design layer is deformed under the pattern formed by the structural layer and the structural layer in its edge regions is reshaped in such a way that its surfaces, in cross-section, converge tangentially.

Abstract: The invention relates to a mounted module (20) for a motor vehicle (1), comprising an optical sensor system (3) that is suitable for a) monitoring a detection area (21) located outside the vehicle (1), b) triggering a signal for starting an authentication process between an ID generator (13) and the motor vehicle (1) if a user (10) is detected in the detection area (21), c) monitoring an actuation area (22) which is located outside the vehicle (1) and differs from the detection area (21), d) providing an operating signal for the vehicle (1) if a user is detected in the actuation area (22).

Abstract: The invention provides gene therapy vectors, such as adeno-associated vims (AAV) vectors, expressing a functional fragment of the miniaturized human micro-dystrophin gene and method of using these vectors to express the fragment of micro-dystrophin in skeletal muscles including diaphragm and cardiac muscle and to protect muscle fibers from injury, increase muscle strength and reduce and/or prevent fibrosis in subjects suffering from muscular dystrophy.

Abstract: The invention relates to an assembly module (20) for a motor vehicle (1), comprising an optical sensor system (30) which can be used a) to monitor a detection area (150) on the outside of the motor vehicle (1) in order to determine the proximity of a user (10), b) to measure the distance to the user (10) within the detection area (150) and c) to release a signal for carrying out an action on the motor vehicle (1) in the event a user (10) is detected in a predefined actuation area (180) within the detection area (150).

Abstract: The invention relates to an assembly module (20) for a motor vehicle, comprising an optical sensor system (30), which can be used to a) monitor a detection area (21) on the outside of the motor vehicle (1) in order to determine the proximity of a user (10), b) in the event a user (10) is detected in the detection area (21), a signal for starting an authentication test between an ID transmitter (13) and an access control system (14) of the motor vehicle (1) is triggered.