MicroRNAs for Cell Line Utilization and Future Therapeutic Application

This technology includes microRNAs for use in cell lines for protein production and potentially future treatments of cancer or diseases related to metabolism. Mmu-miR-466h was identified as a major apoptotic regulator in suspension adapted Chinese Hamster Ovary cells. Mmu-miR-466h was found to have the pro-apoptotic activity by targeting some anti-apoptotic genes for degradation during the exposure of CHO-S cells to the nutrients depleted media.

Ionophores as Treatment for Sickle Cell Disease

This technology includes a method using ionophores to reduce sickling in patients with sickle cell disease. Sickle cell disease is caused by polymerization of a hemoglobin mutant, and the only approved treatment acts by replacing sickle hemoglobin with fetal hemoglobin, thereby increasing the delay time prior to polymerization. This drug is only partially successful because it does not induce fetal hemoglobin synthesis in all cells.

Structure-Based Design of A3 Adenosine Receptor-Selective 2-Arylethynyl (N)-methanocarba Nucleosides for Diagnostic and Therapeutic Uses

This technology includes compounds that are selective agonists of the A3 receptor for the treatment of various disorders such as cancer and autoinflammatory diseases. Structurally, these compounds extend the class of (N)-methanocarba derivatives that are selective agonists of the A3 receptor.

Ribose Derivatives as A3 Adenosine Receptor Modulator for Various Therapeutic Uses

This technology includes a class of A3AR-selective agonists to be used therapeutically to treat a variety of conditions, including chronic pain, cancer, and inflammatory diseases. This class of compounds produced full agonists of the human A3AR of nanomolar affinity that were consistently highly selective (>1000-fold vs. A1AR and A2AAR). The selectivity at mouse A3 receptors is smaller, but the compounds are still effective in vivo in reducing or preventing development of neuropathic pain.

Polyclonal Antibodies to Apolipoprotein L1 for Use in Basic Science Research

This technology includes antibodies to apolipoprotein L1 (ApoL 1) to be used in basic science laboratory studies. ApoL 1 is a protein that is present within cells and circulates as component of high-density lipoprotein. Its functions are not well understood. Recently APOL 1 genetic variants have been shown to be highly associated with kidney disease in African Americans.

Mouse Models for the Study of Gaucher Disease and Therapeutic Development

This technology includes mouse models for both mild and severe Gaucher disease. Gba-L444P and Gba-L444P A456P mice, respectively, carry common gene mutations for milder or severe Gaucher disease, a lysosomal storage disease. Gaucher Disease is caused by mutations in the lysosomal enzyme, glucocerebrosidase. Deficiency of enzyme activity leads to the accumulation of glucosylceramide in liver, spleen, bone, and in the most severe cases, the central nervous system.

Shingosine Kinase 2 (Sphk2) Knock Out Mouse for Neurobiology and Immunology Research

This technology includes a knockout mouse model for Sphingosine kinase 2 (Sphk2) to be used in neurobiology and immunology research studies. Sphingosine kinase 1 and 2 are enzymes that produce sphingosine-1-phosphate, a potent bioactive compound that activates a family of G-protein coupled receptors known as Edg or S1P receptors. Triggering these receptors on cells may have important effects related to inflammation, immunity, cancer, angiogenesis, cell proliferation, adhesion, cardiovascular function, nervous system function and injury responses.

PPTN as a Selective P2Y14 Receptor Antagonist for the Discovery of Treatments of Inflammatory Disorders

This technology includes PPTN which can be used to study treatments of inflammatory diseases. PPTN is currently a useful pharmacological probe that many labs in the field of purinergic signaling are interested in obtaining. The availability of PPTN as a research tool will stimulate basic advances in the field and possibly eventually lead to new treatments. However, PPTN itself is unsuitable for therapeutic applications. Separately, we are working on new and improved antagonists of the P2Y14 receptor.

Mouse Models for the Study of Male Fertility

This technology includes two mouse models to be used in studying male sterility. One mouse is deficient in the full-length protein for STAMP/TtH5. The second is a conditional mutant STAMP mouse that can be used to produce tissues/organs that are deficient in full length STAMP. STAMP represents an intriguing new protein in the study of male fertility. More detailed future studies should identify the precise defect(s) leading to male sterility and may identify other behavioral and developmental consequences, such as a role in the immune system that is suggested by the microarray studies.