Main Menu

The technology discloses composition of compounds that fully antagonize the human P2Y14 receptor, with moderate affinity with insignificant antagonism of other P2Y receptors. Therefore, they are highly selective P2Y14 receptor antagonists. Even though there is no P2Y14 receptor modulators in clinical use currently, selective P2Y14 receptor antagonists are sought as potential therapeutic treatments for asthma, cystic fibrosis, inflammation and possibly diabetes and neurodegeneration.

This technology includes a robust and highly efficient protocol that differentiates induced pluripotent stem cells (iPSCs) exclusively into nociceptors (also called sensory neurons) under chemically defined conditions. The use of hPSCs, including hESCs and iPSCs, holds great promise for disease modeling, drug discovery, and cell therapy. However, efficient and highly reproducible protocols have not been developed for most cell types that are relevant and urgently needed for translational applications.

This technology includes a robust and highly efficient protocol that differentiates human pluripotent stem cells (hPSCs) exclusively into nociceptors (also called sensory neurons) under chemically defined conditions. The use of hPSCs, including hESCs and iPSCs, holds great promise for drug screening, disease modeling, toxicology, and regenerative medicine. However, efficient and highly reproducible protocols have not been developed for most cell types that are relevant and urgently needed for translational applications.

This technology includes a robust and highly efficient protocol that differentiates human pluripotent stem cells (hPSCs) into the developmental precursor of placental cells, the trophectoderm (TE), under chemically defined conditions. The in vitro generation of TE cells holds great promise for modeling diseases of the placenta, drug screening, and cell-based therapies.

This technology includes a method for creating functional, brain region-specific neural spheroids that can be used for disease modeling and therapeutic testing of compounds for neurological diseases. The developed protocol uses somatic cells, including iPSC-derived neurons, as well as astrocytes using means such as 96- or 384-well ultra-low attachment round-bottom plates. Spheroids have been generated using this method that model brain regions such as the ventral tegmental area and prefrontal cortex, which are implicated in Parkinson’s and Alzheimer’s disease.

This technology includes the use of the Anneal-Pool-Ligate-Sequence method (APLS) to quantify the cellular expression of dozens of genes for high throughput chemical library screening. This method is performed by culturing eucaryotic cells in 384-well format microplates, treating the cells with a library of chemicals, and producing cell lysates. Oligodeoxynucleotide (oligo) pairs representing (21) selected genes, and carrying index sequences for each well (384) and microplate (26), are annealed to mRNAs in cell lysates.

Scientists at NCATS have developed a novel Cellular Thermal Shift Assay (CETSA), named “Real-time CETSA” in which temperature-induced aggregation of proteins can be monitored in cells in real time across a range of compound concentrations and simultaneously across a temperature gradient in a high-throughput manner. Real-time CETSA streamlines the thermal shift assay and allows investigators to capture full aggregation profiles for every sample.

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 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 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.