Abstract: The present invention relates to a nucleic acid expression cassette, in particular for the expression of a human liver-specific and/or liver-expressed protein and/or preferably physiologically active domains and/or fragments thereof in a patient suffering from a monogenetic disorder caused by a mutation in the gene coding for the liver-specific and/or liver-expressed protein.

Abstract: In a first aspect, provided herein are chimeric antigen receptors (CAR) composed of at least an extracellular domain, a transmembrane domain and an intracellular domain, the extracellular domain comprises a spacer domain located C-terminally to a ligand-binding-domain also present in the extracellular domain, whereby the spacer domain comprises at least part of the CD34 molecule. Also provided are nucleic acid molecules encoding the same as well as vectors and cells containing the same. The cells include engineered T-cells and NK-cells and derivatives thereof. A pharmaceutical composition comprising the CAR e.g. in form of a vector, a polynucleotide encoding the CAR or the CAR itself and, in addition, cells, cell lines or host cells accordingly are provided. The CAR is useful in adoptive cell therapy. Finally, a method for enrichment or purification of CAR, in particular, of genetically engineered cells, cell lines or host cells expressing the CAR is provided.

Abstract: Disclosed herein are compositions and methods for the expression of a gene of interest. The disclosed methods may employ codon-optimization and introduction of non-endogenous restriction sites for efficient expression of a gene. The methods may further employ introduction of a gene variant of interest, such that the disclosed methods, compositions, and systems may be used to determine the significance of a variant of interest. Further disclosed are compositions, systems, and methods for the characterization of gene variants, and other mutations that may impact the function of the protein of interest.

Abstract: The present invention relates to conjugated gold nanoparticles, preferably for the use in the treatment of a monogenetic disorder resulting from a mutation in a gene coding for a liver-specific and/or liver-expressed protein, comprising laser-ablated gold nanoparticles, polyethylenimine (PEI) and/or derivatives and/or salts thereof and a nucleic acid molecule. Furthermore, the present invention relates to the use of such gold nanoparticles, a method for the preparation of conjugated gold nanoparticles, a nanoparticle-based delivery system and the use of such delivery system. In addition, the present invention relates to a method for transfection of target cells, a transfected target cell as well as a vector for the expression of a liver-specific and/or liver-expressed protein.

Abstract: The present invention relates to a nucleic acid expression cassette, in particular for the expression of a human liver-specific and/or liver-expressed protein and/or preferably physiologically active domains and/or fragments thereof in a patient suffering from a monogenetic disorder caused by a mutation in the gene coding for the liver-specific and/or liver-expressed protein.

Abstract: The invention discloses in vitro methods and a system for determining a predisposition of a subject for developing a cancer on the basis of analyzing a sample of the subject for an alteration of at least one allele of the RAD51C gene. Further disclosed are in vitro methods for assessing clinical features or a pathological progression of a cancer and for assessing at least one RAD51C gene alteration in a cell. In addition a kit for determining a predisposition of a subject for developing a cancer, and certain uses of oligonucleotides for determining the presence of at least one mono-allelic germ-line mutation of the RAD51C gene.

Abstract: In a first aspect, provided herein are chimeric antigen receptors (CAR) composed of at least an extracellular domain, a transmembrane domain and an intracellular domain, the extracellular domain comprises a spacer domain located C-terminally to a ligand-binding-domain also present in the extracellular domain, whereby the spacer domain comprises at least part of the CD34 molecule. Also provided are nucleic acid molecules encoding the same as well as vectors and cells containing the same. The cells include engineered T-cells and NK-cells and derivatives thereof. A pharmaceutical composition comprising the CAR e.g. in form of a vector, a polynucleotide encoding the CAR or the CAR itself and, in addition, cells, cell lines or host cells accordingly are provided. The CAR is useful in adoptive cell therapy. Finally, a method for enrichment or purification of CAR, in particular, of genetically engineered cells, cell lines or host cells expressing the CAR is provided.

Abstract: The invention relates to a modified human CYP4B1 protein and a nucleic acid encoding for the modified human CYP4B1 protein. The invention further relates to an expression vector comprising a nucleic acid encoding for a modified human CYP4B1 protein, as well as to a cell comprising a modified CYP4B1 protein. In addition, the invention relates to a modified human CYP4B1 protein for use in treating transplantation induced graft-versus-host-disease (GvHD), and to 4-ipomeanol (4-IPO) for use in killing a cell.

Abstract: The invention discloses in vitro methods and a system for determining a predisposition of a subject for developing a cancer on the basis of analyzing a sample of the subject for an alteration of at least one allele of the RAD51C gene. Further disclosed are in vitro methods for assessing clinical features or a pathological progression of a cancer and for assessing at least one RAD51 C gene alteration in a cell. In addition a kit for determining a predisposition of a subject for developing a cancer, and certain uses of oligonucleotides for determining the presence of at least one mono-allelic germ-line mutation of the RAD51C gene.

Abstract: The disclosure of the present application provides foamyvirus vectors, as well as methods and kits involving the same. In at least one embodiment, a plurality of recombinant vectors comprises an expression sequence encoding at least one component of a foamyvirus particle, wherein at least one codon of the expression sequence is optimized for expression in homo sapiens.

Abstract: The invention relates to a method for isolation of target molecules from a nucleic acid population.