Abstract: Disclosed are nitrosourea and other compounds, pharmaceutical composition, and methods of treating cancers that are MGMT deficient regardless of their MMR status and particularly compounds, pharmaceutical compositions, and methods of treating cancers that are both MGMT and MMR deficient.

Abstract: In various aspects and embodiments the invention provides a method of treating or preventing a cancer in a mammalian subject in need thereof, the method comprising administering to the subject a therapeutically effective amount of at least one compound selected from the group consisting of a DNA repair inhibitor, a DNA strand break repair inhibitor, and a homologous recombination (HR) repair inhibitor, wherein cells in the cancer comprise a fumarate hydratase (FH) and/or succinate dehydrogenase (SDH) mutation.

Abstract: The present invention provides a method of treating cancer in a subject, the method comprising administering to the subject at least one nicotinamide phosphoribosyltransferase (NAMPT) inhibitor, thereby treating the cancer, wherein protein phosphatase Mg2+/Mn2+ dependent 1D (PPM1D) is elevated in the cancer.

Abstract: In various aspects and embodiments the invention provides a method of treating or preventing a cancer in a mammalian subject in need thereof, the method comprising administering to the subject a therapeutically effective amount of at least one compound selected from the group consisting of a DNA repair inhibitor, a DNA strand break repair inhibitor, and a homologous recombination (HR) repair inhibitor, wherein cells in the cancer comprise a fumarate hydratase (FH) and/or succinate dehydrogenase (SDH) mutation.

Abstract: The invention includes compositions and methods for treating or preventing a cancer in a subject. In one aspect the invention provides methods of administering to a subject suffering from a cancer with cells containing an IDH1 or IDH2 mutation, at least one compound comprising a DNA repair inhibitor. The invention also provides methods of treating a cancer with 2 HG, a derivative of 2-HG, any variant and any mixtures thereof. In one aspect the invention provides a pharmaceutical composition comprising an anti-tumor effective mount of at least one compound selected from the group consisting of 2-hydroxyglutarate (2-HG), a derivative of 2-HG, any variant and any mixtures thereof.

Abstract: Compositions for targeted mutagenesis of cell surface receptors for HIV and methods of their use are provided herein. The compositions include triplex-forming molecules that bind to duplex DNA in a sequence specific manner at target sites to form triple-stranded structures. The triplex-forming molecules can be triplex-forming oligonucleotides (TFOs) or peptide nucleic acids (PNAs). The triplex-forming molecules are useful to induce site-specific homologous recombination in mammalian cells when used in combination with donor oligonucleotides. The triplex-forming molecules target sites within or adjacent to genes that encodes cell surface receptors for human immunodeficiency virus (HIV). This binding stimulates homologous recombination of a donor oligonucleotide to cause mutations in HIV cell surface receptor genes that result in one or more deficiencies in the ability of the encoded receptor to bind to HIV and allow its transport into the cell.