Abstract: A method for analysis of omics data related to samples derived from a human, animal or disease model is disclosed. The method comprises the steps of: a) Determining a plurality of latent spaces by inputting the omics data into a plurality of omics neural networks in parallel and achieving a plurality of latent spaces each comprising a set of latent factors by performing the calculations of the plurality of omics neural networks in a data processing unit, wherein the output of the omics neural networks each provides a respective latent space; b) Consolidating the plurality of latent spaces into one reproducible latent space, wherein the step of consolidating is processed by a data processing unit; wherein the consolidated latent factors represent characteristics of the samples to be analyzed.

Abstract: A method for modifying double stranded DNA (dsDNA) employing an RNA guided DNA endonuclease to generate two double strand breaks in the dsDNA molecule to be modified, and replacement of the sequence positioned between the double strand breaks with a substitute DNA sequence using the non-homologous end joining (NHEJ) pathway, and corresponding kits and compositions for modifying double stranded DNA molecules.

Abstract: A nucleic acid molecule encoding an antibody or antibody fragment, wherein the antibody or antibody fragment binds an epitope of the extracellular domain of CD269 (BCMA), a host cell comprising the nucleic acid molecule and a composition comprising the host cell.

Abstract: A method for generating a three-dimensional neuromuscular organoid in vitro includes providing a first cell culture that includes disease-specific neuromesodermal progenitor cells, 30% to 90% of which co-express BRACHYURY/SOX2 and 10% to 70% of which co-express TBX6. The disease-specific neuromesodermal progenitor cells are cultivated in a first differentiation medium chosen from the group consisting of i) a non-supplemented serum-free cell culture medium and ii) a serum-free cell culture medium supplemented with at least one of a ROCK inhibitor, an activator of a growth factor signaling pathway, and an activator of an insulin signaling pathway. The first differentiation medium is replaced by a second differentiation medium within 1 to 3 days after cultivation start. The second differentiation medium is replaced by a non-supplemented serum-free cell culture medium within another 1 to 3 days. A three-dimensional neuromuscular organoid is obtained from the non-supplemented serum-free cell culture medium.

Abstract: A composition comprising a cell or a population thereof, wherein the cell comprises a polynucleotide encoding an antibody or a fragment thereof that binds CD269 (BCMA), wherein the binding to CD269 (BCMA) disrupts the interaction between CD269 and its native ligands (BAFF and APRIL). Also disclosed is treatment of plasma cell-mediated diseases such as multiple myeloma and autoimmune diseases.

Abstract: The present invention refers to hyperactive variants of a transposase of the transposon system Sleeping Beauty (SB). The invention further refers to corresponding nucleic acids producing these variants, to a gene transfer system for stably introducing nucleic acid(s) into the DNA of a cell by using these hyperactive variants of a transposase of the transposon system Sleeping Beauty (SB) and to transposons used in the inventive gene transfer system, comprising a nucleic acid sequence with flanking repeats (IRs and/or RSDs). Furthermore, applications of these transposase variants, the transposon, or the gene transfer system are also disclosed such as gene therapy, insertional mutagenesis, gene discovery (including genome mapping), mobilization of genes, library screening, or functional analysis of genomes in vivo and in vitro. Finally, pharmaceutical compositions and kits are also encompassed.

Abstract: An isolated chimeric antigen receptor polypeptide (CAR), wherein the CAR comprises an extracellular antigen-binding domain, comprising an antibody or antibody fragment that binds a human CXC chemokine receptor type 5 (CXCR5) protein. Also disclosed is a nucleic acid molecule encoding the CAR of the invention, a genetically modified immune cell, preferably a T cell, expressing the CAR of the invention and the use of said cell in the treatment of a medical disorder associated with the presence of pathogenic cells expressing CXCR5, preferably pathogenic mature B cells and/or memory B cells, and/or pathogenic T cells and/or T follicular helper cells, in particular mature B cell non-Hodgkin's lymphoma (B-NHL), T cell non-Hodgkin's lymphoma, or autoantibody-dependent autoimmune disease, preferably selected from systemic lupus erythematosus (SLE) or rheumatoid arthritis.

Abstract: The present invention pertains to novel high avidity antigen recognizing constructs, such as antibodies or T cell receptors, which specifically bind to the melanoma associated antigen (MAGE) A1. The constructs of the invention are particularly useful for the diagnosis, prevention or therapy of tumorous diseases which are characterized by the specific expression of the MAGE-A1 antigen. Furthermore provided are nucleic acids, vectors and host cells—such as CD4 or CD8 positive T cells—which encode, comprise or present the antigen recognizing constructs of the invention. The invention thus provides new approaches for immune therapy, specifically adoptive T cell therapy, for treating cancer.

Abstract: The invention relates to a system and method for introducing DNA into cells. In particular, the invention relates to a method for introducing single or multiple copies of a DNA sequence or gene of interest into a cell comprising providing: a) a “copy and paste” transposase; and b) a construct comprising a DNA sequence or gene of interest flanked by a “copy and paste” transposon terminal sequence, such as an LTS or RTS. A novel “copy and paste” transposon of the Helitron family is described along with systems for using the corresponding transposase in methods for introducing DNA into cells, for example, to generate cell lines for use in protein production, cell and gene therapy or as reference standards.

Abstract: Chemical compounds that are useful as inhibitors of mechanotransduction in the treatment of pain and modulation of touch perception and topical administration of the compounds described herein in the treatment of pain and modulation of touch perception.

Abstract: An in vitro method for identifying, isolating and/or enriching primate naive pluripotent stem cells, the method including analyzing transcription of a type 7 long terminal repeat (LTR7) nucleic acid sequence of a type H human endogenous retrovirus (HERVH) (LTR7/HERVH-associated transcription), and identifying, isolating and/or enriching primate naive pluripotent stem cells based on LTR7/HERVH-associated transcription, wherein LTR7/HERVH-associated transcription is a marker for primate naive pluripotent stem cells. An isolated in vitro population of primate naive pluripotent stem cells is obtained by the method, wherein in the cells LTR7/HERVH-associated transcription is elevated in comparison to control cells, wherein control cells are primed pluripotent stem cells or differentiated cells.

Abstract: A method for generating a three-dimensional neuromuscular organoid in vitro is disclosed.

Abstract: A cosmetic method for modulating pigmentation in a subject includes administering a modulator of angiotensin-converting enzyme 2 (ACE2 modulator) to the subject. The ACE2 modulator can be an inhibitor of angiotensin-converting enzyme 2 (ACE2 inhibitor), in which case, the ACE2 inhibitor can be administered to increase pigmentation in the subject. The ACE2 modulator can also be an activator of angiotensin-converting enzyme 2 (ACE2 activator), in which case the ACE2 activator can be administered to decrease pigmentation in the subject. The treatment of inflammatory skin disease can also be achieved by inhibition of angiotensin-converting enzyme 2.

Abstract: A method for inhibiting, reducing and/or reducing the risk of cancer metastasis in a subject, including identifying a subject as in need of inhibiting, reducing and/or reducing the risk of cancer metastasis, by identifying the subject as having a cancer with an elevated or up-regulated level of S100A4 transcription compared to a level in non-oncogenic cells, and administering to the subject a therapeutically effective amount of niclosamide or a niclosamide derivate, thereby inhibiting or reducing S100A4 transcription, so as to inhibit, reduce and/or reduce the risk of cancer metastasis, wherein 15 to 400 mg niclosamide or niclosamide derivate is administered per kg body weight of the subject (mg/kg), 1 or 2 times daily.

Abstract: A microscope-autofocus device for feedback-controlling a focal position of an imaging system of a microscope apparatus, wherein the imaging system includes a microscope objective, a monitoring beam source for creating a monitoring beam, a detector device for detecting a drift variation of an axial objective distance between the microscope objective and a sample by sensing the monitoring beam directed through the imaging system to the sample and reflected by the sample, and a feedback loop device for controlling the imaging system in dependency on the detected objective distance variation of the microscope objective.

Abstract: The present invention relates to a method for the quantification of seeding (?t50) of an amyloidogenic aggregate, methods for assessing the risk for development, predicting the onset or assessing the progression of a polyQ disease, and a method for identifying compounds that inhibit mHTT seeding activity (HSA) in vitro. Further, uses of fluorophore-bearing polyQ proteins, particularly mutant N-terminal huntingtin fragments comprising exon-1 and related soluble protein constructs are provided.

Abstract: The present invention relates to compounds according to general formula (I) which are metabolically robust analogues of bioactive lipid mediators derived from omega-3 polyunsaturated fatty acids (n-3 PUFAs). The present invention further relates to compositions containing one or more of these compounds and to the use of these compounds or compositions for the treatment or prevention of cardiovascular diseases.

Abstract: The invention relates to a xanthine derivative defined by chemical formula I or a salt thereof, its use as a medicament, especially for use in the treatment of serotonin-related diseases or disorders, and a pharmaceutical preparation comprising the xanthine derivative. The novel xanthine compounds are capable of inhibiting tryptophan hydroxylases (TPH) involved in the biosynthesis of serotonin and are effective in influencing the serotonin level in the body.

Abstract: A method is for treatment of a disease associated with genotoxic stress-induced inhibitor of nuclear factor-?B kinase/nuclear factor kappa-light chain enhancer of activated B cells (IKK/NF-?B) signaling. The method can include administering a compound to a subject having a cancer exhibiting genotoxic stress induced IKK/NF-?B activation.

Abstract: The present invention relates to the field of immunotherapy, in particular, to adoptive T cell therapy, T cell receptor (TCR) gene therapy and vaccination. The invention provides a method for preparing a nucleic acid encoding the TCR alpha chain construct (TRA) and TCR beta chain construct (TRB) of a TCR construct specific for an epitope from an antigen presented on major histocompatibility complex (MHC), comprising contacting T cells isolated from a donor with a library of artificial antigen presenting cells (APC) comprising cells expressing all MHC I or MHC II alleles present in the donor, preferably, in K562 cells. The TCR construct can be expressed in a T cell, which is useful for adoptive T cell therapy, e.g., of cancer, viral infections or autoimmune diseases. The invention further provides a method for identifying the epitope recognized by said TCR. Immunogenic epitopes recognized by said TCRs can be used to develop vaccine formulations to induce antigen-specific T cell immunity in patients.