Disclosed herein are polynucleic acid molecules, plasmids, vectors, compositions, methods, and kits for expressing a target protein. In some instances, also described herein are polynucleic acid molecules, plasmids, vectors, compositions, methods, and kits for enhancing the expression of a target protein.

Described herein are compositions and methods for treating, inhibiting or ameliorating X linked agammaglobulinemia (XLA) in subjects that have been identified or selected as being ones that would benefit from a therapy to treat, inhibit, or ameliorate XLA. Exemplary embodiments include constructs and methods for gene therapy, which restore or increase BTK expression.

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.

Disclosed herein are vector systems for expression of polypeptides in eukaryotic cells; and methods of obtaining high-level expression of polypeptides in a eukaryotic cell. Methods and compositions for obtaining stable, long-term expression of recombinant polypeptides are also provided

The present invention relates to a polynucleotide comprising at least one promoter and an S/MAR element, wherein said S/MAR element is located downstream of said promoter in the 3 UTR of the transcription unit and wherein the said S/MAR element is flanked by a 5 splice donor site and a 3 splice acceptor site; the present invention further relates to a composition comprising said polynucleotide, and to the polynucleotide for use in medicine and for use in treating genetic disease.

The present invention relates to the field of self-replicating non-integrative episomal vertebrate expression vectors useful for in gene therapy, ex vivo cell therapy, stem cell therapy, and more particularly, for improving the expression of vector encoded antigens or therapeutic genes. Such recombinant DNA molecules are useful in biotechnology, transgenic organisms, gene therapy, stem cell therapy, therapeutic vaccination, agriculture and DNA vaccines. More specifically, relates to a polynucleotide comprising at least one promoter and an S/MAR element, wherein said S/MAR element is located downstream of said promoter and wherein the nucleic acid sequence of said S/MAR element (S/MAR sequence) comprises at least 3 sequence motifs ATTA (SEQ ID NO:1) per 100 nucleotides over a stretch of at most 200 nucleotides; the present invention further relates to a composition and to a host cell comprising said polynucleotide, and to the polynucleotide for use in medicine and for use in treating genetic disease. The present invention also relates to a kit and to a device comprising said polynucleotide, and to methods and uses related to the polynucleotide.

The invention is directed to a more efficient lentiviral vector comprising a nucleic acid sequence encoding a human -globin protein or a human -globin protein, which is oriented from 5 to 3 relative to the lentiviral genome. The invention also provides a composition and method utilizing the lentiviral vector.

In the present invention, the inventors have generated a Tet-On all-in-one construct that tightly regulate transgene expression in human stem cells using the original TetR repressor. By using appropriate promoter combinations and shielding the integrative construct with the Is2 insulator, they have constructed the Lent-On-Plus Tet-On system that achieves efficient transgene regulation in human multipotent and pluripotent stem cells. The generation of inducible stem cell lines with the Lent-ON-Plus system did not require selection or cloning, and transgene regulation is maintained after long-term cultured and upon differentiation toward different lineages. The present invention thus offers a solution to the need to improve transgene expression systems in stem cells.

MiniVectors and compositions containing MiniVectors that target ovarian cancer genes selected from FOXM1, AKT, CENPA, PLK1, CDC20, BIRC5, AURKB, CCNB1, CDKN3, BCAM-AKT2, CDKN2D-WDFY2, SLC25A6, CIP2A, CD133, ALDH1A1, CD44, SALL4, and/or PRDM16, alone or in any combination, are provided, along with uses in the treatment of ovarian cancer.

The invention is directed to a more efficient lentiviral vector comprising a nucleic acid sequence encoding a human -globin protein or a human -globin protein, which is oriented from 5 to 3 relative to the lentiviral genome. The invention also provides a composition and method utilizing the lentiviral vector.