A Novel High-Throughput Assay for Identifying Zike Virus NS2B-NS3 Protease Inhibitors

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.

Repurposed Use of the Alkaloids Emetine and Cephaeline to Treat Zika Virus Infection

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.

Repurposing CDK Inhibitors for the Treatment of Zika Virus Infection

This invention includes the discovery and use of a group of CDK inhibitors that were found during a drug repurposing screen designed to find compounds that inhibit Zika virus caused cell death. The identified CDK inhibitors have all previously been used in clinical trials for other diseases, potentially reducing the long time course needed for new drug discovery and development.

Novel Codon-Optimized MUT Gene Therapeutic for Methylmalonic Acidemia (MMA)

Methylmalonic Acidemia (MMA) is a metabolic disorder characterized by increased acidity in the blood and tissues due to toxic accumulation of protein and fat by-products resulting in seizures, strokes, and chronic kidney failure. A significant portion of MMA cases stem from a deficiency in a key mitochondrial enzyme, methylmalonyl-CoA mutase (MUT), required to break down amino acids and lipids. Currently, there are no treatments for MMA and the disease is managed primarily with dietary restriction of amino acid precursors and liver-kidney transplantation in severe cases.

Fibroblast Cell Lines (with L444P/RecNci1 Genotype) for the Screening of Small Molecules for Gaucher Disease Treatment

This technology includes two human fibroblast cell lines to be used to study the defects in GBA1 gene and protein and to screen small molecules for involvement in Gaucher disease. Glucocerebrosidase (GBA1 or GCase or beta-glucosidase) is a lysosomal enzyme, responsible for breakdown of a fatty material called glucocerebroside (or glucosyl ceramide). Deficiency or malfunction of GBA1 leads to the accumulation of insoluble glucocerebrosides in tissues, which is a major symptom of Gaucher disease.

Staphylococcus Epidermidis Isolates from Human Skin Samples for Use as Clinical Molecular Markers

This technology includes a catalog of commensal and pathogenic staphylococci from human skin for utilization as clinical molecular markers of skin conditions and infections. The study of microbial diversity of human skin in both healthy and disease states is important to develop tools to track infections, outbreaks, and multi-drug resistant organisms, particularly in atopic dermatitis, eczema and other microbial-associated infections. Commensal skin S. epidermidis have an open pan-genome and show considerable diversity between isolates.

In-vivo System to Interrogate the Functions of Mucous Membranes and Identify Mucin/Glycan Mimetics and JAK/STAT Inhibitors for the Treatment of Diseases of the Oral Cavity and Digestive Tract

This technology includes a Drosophila mutant strain that can be used as an in vivo model for diseases of the oral cavity and digestive tract (Sjogren's syndrome, colitis, colon cancer, inflammatory bowel disease), where the mucous membrane is disrupted or non-functional. This mutant lacks a mucous membrane and displays epithelial cell damage, uncontrolled cell proliferation and the up-regulation of conserved signaling pathways (JAK/STAT).

Replicating RNA Vaccine For Crimean-Congo Hemorrhagic Fever Virus

Crimean-Congo hemorrhagic fever (CCHF) is a deadly hemorrhagic fever having a high mortality rate. The disease results from infection of an individual by Crimean-Congo hemorrhagic fever virus (CCHFV), which is a tick-borne bunyavirus endemic in Southern and Eastern Europe, Africa, the Middle East, and Asia. Geographically, case distribution is consistent with the range of Hyalomma genus ticks, the main reservoir of CCHFV, and is likely to expand due to climate change. Humans may be infected from tick bites, through contact with infected animals or animal tissue.

Hybridomas Producing Antibodies to Neuraminidase for Influenza A (H3N2) Diagnostics, Vaccine, and Therapeutic Development

Influenza A and B viruses can cause seasonal flu epidemics ― commonly known as the “flu season” ― and infect the nose, throat, eyes, and lungs in humans. Typically, flu seasons that are dominated by influenza A (H3N2) virus activity have higher associated hospitalizations and deaths in at-risk groups, such as people ages 65 and older and young children. Influenza A (H3N2) virus can also cause respiratory disease in animals, such as canines and swine.

Codon Deoptimized (CD) Poliovirus Seed Strains for Use in an Inactivated Poliovirus Vaccine

Polio is a disabling and potentially fatal infectious disease. Sabin Oral Poliovirus Vaccine (OPV) and Salk Inactivated Poliovirus Vaccine (IPV) have been crucial in the global poliovirus eradication efforts and substantial decrease in disease incidence rates. However, recent findings showed that Sabin OPV strains, due to their genetic instability, may revert to virulence and spread among communities, resulting in circulating vaccine-derived poliovirus (cVDPV). Salk IPV, which is made by inactivating live poliovirus,