Abstract: The invention relates to methods of treating or preventing acquired or congenital mitochondrial diseases or disorders, including treating cell proliferation related disorders or diseases, such as cancer. The invention further relates to pharmaceutical compositions for treating such disorders or diseases.

Abstract: A method of treating cancer in a subject comprising the step of administering to the subject in need thereof an effective amount of a combination of a compound that binds a nuclear export signal (NES inhibitor) on topoisomerase II? and a topoisomerase inhibitor. Twenty small molecule inhibitors (SMI) that bind to the two nuclear export sequences (NES) topo II? have been identified from the NCI database using computer-generated molecular modeling. These SMI will improve the effectiveness of topo II directed therapeutics, particularly in the treatment of diseases such as multiple myeloma (MM). In vitro apoptosis assays indicate that these drugs may be effective as single agents or in combination with currently used cancer drugs that target topo II.

Abstract: The invention relates to methods for inducing male contraception and to methods of treating cell proliferation related disorders or diseases, such as cancer. The invention further relates to pharmaceutical compositions for inducing male contraception and for treating cell proliferation related disorders or diseases.

Abstract: The invention features compositions and methods that are useful for the treatment of neoplasia (e.g., breast cancer) by increasing DNA damage and reducing base excision repair (BER).

Abstract: The invention provides compositions and methods for the treatment of neoplasias that are cytotoxic to neoplastic cells or that modulate JAZ expression, subcellular localization, or biological activity.

Abstract: The invention relates to phosphorylation inhibitor compounds and methods of identifying and using them. The invention further relates to pharmaceutical compositions and methods for treating cell proliferative disorders, especially cancer.

Abstract: The subject application provides small compounds that are able to suppress autophagy in various cells. These compounds are useful in augmenting the existing treatments of various cancers and microbial/parasitic infections. Thus, the subject application also provides methods of treating various types of cancers and microbial/parasitic infections. Also provided by the subject application are methods of suppressing the expansion of autophagosomes within cells or individuals and inhibiting the lipidation of autophagy-related protein 8 (Atg8).

Abstract: The invention features compositions and methods that are useful for the treatment of neoplasia by reducing base excision repair (BER). Such compositions are useful, for example, for enhancing the efficacy of known chemotherapeutics, such as DNA alkylating agents. In particular, the invention features agents that mimic the interaction of APC with pol-?. Such agents reduce the activity of long patch- and single nucleotide-base extension repair pathways.

Abstract: The invention features compositions and methods that are useful for treating or preventing AAT deficiency and associated conditions. In addition, the invention provides methods for identifying compounds useful for treatment of AAT deficiency and associated conditions.

Abstract: The invention relates to cyclin dependent kinase inhibitor compounds and methods of identifying and using them. The invention further relates to pharmaceutical compositions for treating cell proliferative disorders, especially cancer.

Abstract: The present invention provides compositions and methods useful in the treatment and/or prevention of ophthalmic conditions and diseases, such as retinitis pigmentosa, that are dependent upon or related to misfolded opsin proteins in vivo. In addition, screening assays for agents useful in such treatment methods are described.

Abstract: The invention relates to protein binding inhibitor compounds and methods of identifying and using them. The invention further relates to pharmaceutical compositions and methods for treating a variety of diseases and disorders, including cell proliferative disorders, especially cancer.

Abstract: PKCiota inhibitor 1H-imidazole-4-carboxamide, 5-amino-1-[2,3-dihydroxy-4-[(phosphonooxy)methyl]cyclopentyl]-,[1R-(1?,2?,3?,4?)], (ICA-1), targets a unique sequence (amino acid residues 469-475) in the catalytic domain of PKCiota and inhibits PKCiota activity. The data shows suprising and unexpected ability of ICA-1 to selectively inhibit the proliferation of cells that overexpress PKCiota.