This technology includes a codon optimized expression vector for the high expression and production of RLIP-76 which can be used to provide protection from radiation. RLIP-76 is a multifunctional membrane protein that transports glutathione conjugates of electrophilic compounds outside the cell. The sequence was generated with codon bias alterations, reduction of secondary structure, lowering of GC content, and removal of cryptic elements that could affect expression in E.coli.

This technology includes a group of eight AMPK activating compounds to be further developed for the treatment of Niemann Pick Type C (NPC) disease. Through the recent molecular biology and pharmacological experiments, we have identified the cyclodextrin which directly binds to beta-subunits of AMP-activated protein kinase (AMP), resulting in subsequently activations of AMPK and AMPK linked autophagy, and restoration of autophagy function that is impaired in the NPC cells.

This technology includes a precise measurement assay of cellular FMRP levels in patients, which can assist in the diagnosis and assess the severity of Fragile X syndrome (FXS). FXS is an X-linked disorder that produces intellectual disability, cognitive impairment, epilepsy, depression and anxiety. FXS is caused by mutations in the Fragile X Mental Retardation-1 (FMR1) gene that result in the absence or a loss of function of its protein product, FMRP.

This technology includes the development of devices capable of real-time evaluation of metabolite levels for the treatment of numerous metabolic disorders, including hyperammonemia and aminoacidopathies. Currently, the monitoring of metabolite levels is done in a hospital setting with specialized mass spectrometry instrumentation. As a consequence, susceptible patients who are undergoing a crisis need to visit the hospital for testing to determine if there is a metabolite disturbance.

This technology includes the generation and use of human induced pluripotent stem cell (iPSC) lines that can be used to study and screen potential therapeutics for lysosomal storage diseases (LSDs). LSDs are a group of 50 genetic disorders caused by mutations in the genes encoding lysosomal enzymes and proteins. Although various therapeutic approaches exist, most cases of LSDs are not effectively treated due to a lack of therapeutics (including stem cells and recombinant proteins).

This technology includes the use of novel lactate dehydrogenase (LDH) inhibitors, including WO2018005807A1, for the treatment of primary hyperoxalurias (PHs). PHs are rare autosomal recessive disorders caused by overproduction of oxalate, leading to recurrent calcium oxalate kidney stone disease, and in some cases end-stage renal disease. One potential strategy to treat PHs is to reduce the production of oxalate by diminishing the activity of LDH, the proposed key enzyme responsible for converting glyoxylate to oxalate.

This technology includes the use of a novel formulation for an engineered version of Fibroblast Growth Factor 1 (FGF1), TTHX1114, that can be used to accelerate regeneration of the corneal endothelium after surgical lesions. FGFs are well-established regulators of migration and proliferation of corneal endothelial cells (CECs).

This invention includes a novel high-throughput assay to identify orthosteric inhibitors blocking the Zika virus NS2B-NS3 protease. Pathogenic flaviviruses, including Zika, require the NS2B-NS3 protease for viral replication. There is currently an unmet need for specific antiviral therapeutics against the Zika virus. Preliminary screening using the NCGC Pharmaceutical Collection library identified a group of drugs including temoporfin, erythrosin B, niclosamide, and nitazoxanide that can significantly inhibit the interactions between NS2B and NS3.

This technology includes the use of two related compounds, Emetine and Cephaeline, as a potent inhibitor of the Zika virus (ZIKV). Emetine and it's analog Cephaeline were identified in a high-throughput assay aimed at identifying anti-ZIKV compounds. Both Emetine and Cephaeline are potent inhibitors of ZIKV infection in cell culture, and Emeline is a potent inhibitor of ZIKV infection in a live mouse model.

This technology includes a novel computational algorithm and software implementation to map and display biological pathways and their relationship on the surface of a globe in a three-dimensional space. Currently, biological pathways and genes are represented as two-dimensional networks, which is not effective for displaying complicated relationships between pathways and genes.