Abstract: Disclosed are compositions and methods for treating a disease or disorder such as cancer in a subject in need thereof. In some embodiments, the methods include administering to the subject a vector that has a first nucleic acid sequence encoding a promoter operably linked to each of a second nucleic acid sequence encoding a therapeutic polypeptide, and a third nucleic acid sequence encoding a peptide domain that is stabilized when phosphorylated by kinase activity in a target cell and/or tissue. In some embodiments, the target cell and/or tissue can be a cell and/or tissue undergoing a stress response. In some embodiments, the target cell and/or tissue can be a cell and/or tissue in which a CK2 kinase is active. The kinase activity can be elevated extracellular regulated kinase (ERK) activity, p38 MAP kinase activity, and/or CK2 activity.

Abstract: The present application discloses compositions and methods useful for diagnosing and treating nucleotide repeat disorders. An in silico analysis indicates a striking and specific miR-NRD homology. Validated miR target prediction software was used to assess each NRD nucleotide repeat expansion for potential miR homology. The striking degree of matched homology and the one-to-one expansion-to-miR ratio strongly support their relevance to the corresponding NRD and serve as a basis for therapeutic treatments. It is also disclosed herein that NETO is involved in mediating C9-repeat glutamate excitotoxicity, thus representing a novel therapeutic target.