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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.

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.

This technology includes mice which are intended to be bred with mice that have the tetracycline-response element promoter driving a gene of interest, for the study of kidney diseases. When the dual transgenic mice are fed tetracycline or doxycycline, the transgene is activated in glomerular podocytes.

This technology includes the administration of the antiglucocorticoid mifepristone for the treatment of diabetes. Administration of mifepristone (50 mg orally every 6 hours) for one week improves hepatic and adipose insulin resistance in men and women with pre-diabetes or mild type 2 diabetes mellitus.

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.

This technology includes a series of small molecule TRHR agonists for the treatment of cancer-related fatigue. In particular, PTLS-04 and PTLS-11 have shown excellent TRHR agonist activities in mouse and human cells and have improved metabolic profile compared to TRI I and taltirelin. Additional work is being done to further optimize the series by designing and synthesizing additional compounds.

This technology includes three new chemical entities discovered for antibacterial and antifungal activities. The compounds are novel tetramic acid containing polyketides obtained from two different Amycolatopsis strains. Their planar structures and relative stereochemistry were elucidated by 1D and 2D NMR methods, including 1H-1H and 13C-13C COSY, TOCSY, HSQC, HMBC and ROESY. Whole genome sequencing of these two strains revealed a 158 kb biosynthetic gene cluster (BGC) containing a 23-module, mixed NRPS-PKS pathway responsible for their biosynthesis.

This technology includes a sphingosine kinase 1 (Sphk1) knockout mouse model for use in developmental biology research. Sphingosine-1-phosphate (S1P) is synthesized from sphingosine and ATP by the action of sphingosine kinase, and activates cell signaling. Two sphingosine kinases, SPHK1 and SPHK2, have been identified. To study the physiological function of SPHK1, Sphki null mice were generated. The mice were viable, fertile, with no obvious abnormalities. Total SPHK activity in most tissues was substantially reduced, suggesting the presence of other sphingosine kinases.

This technology includes the use of the (N)-methanocarba phosphonate analogues of 5’-AMP as cardioprotective agents for use in conditions such as cardiomyopathy and heart failure. We previously found a compound, MRS2339 (a phosphate derivative that can be slowly cleaved in vivo and lose potency), which activates the appropriate receptors and is protective in models of heart failure in several species (mouse, dog). MRS2339 is a phosphate derivative that can be slowly cleaved in vivo and lose potency. We now extend this technology to more stable derivatives, i.e.

This technology includes a new class of oligomeric molecules called thyclotides for diagnostic and therapeutic development. Thyclotides is described where chiral tetrahydrofuran (THF) diamine units are linked together with alternating glycines, and nucleobases are attached to this backbone as sidechains. The thyclotide sequence consists of a series of nucleobases similar to that of a nucleic acid sequence. Thyclotides are easily synthesized and purified with excellent solubility in water. Thyclotide sequences bind to complementary DNA and RNA sequences with very strong affinity.