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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 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.

This technology includes two human fibroblast cell lines 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. Gaucher disease is a rare and heterogeneous disorder, caused by inherited genetic mutations in GBA1.

The invention provides two vascularized, multi-chip models for the alveoli and the small airway. Both models comprise a perfusable three-dimensional (3D) microvascular network consisting of human primary microvascular endothelial cells, fibroblasts, and pericytes with a differentiated lung epithelial layer exposed at the air-liquid interface (ALI) on top, built on a high-throughput, 64-chip microfluidic plate platform. The platform does not require the support of a permeable membrane and the epithelial cells are directly seeded on the perfused microvascular network.

This technology relates to a software package called NIMH DAO Toolbox that uses multithreading and a unique buffer structure to shorten gaps in sample readouts. Data acquisition devices running in continuous sampling mode collect data samples at a given sampling rate. The samples are typically stored in a memory buffer and read out at a regular interval. If the sampling rate is short enough, there can be a gap between the time the first sample is acquired and the time that sample is available to the user. This gap is typically on the order of tens of milliseconds.

This technology relates to a closed-loop controller that is being developed as a phone app for emotional self-regulation in real-time. There is a significant association between emotion dysregulation and symptoms of depression, anxiety, eating pathology, and substance abuse, affecting millions worldwide. Consisting of a closed-loop controller that adjusts reward values in real-time according to individual mood response, the Mood Machine Interface technology compensates for adaptation to stimuli over time allowing it to generate substantial mood changes in the user.

This technology includes the combination of an induced pluripotent stem cell (iPSC) line that can inducibly be differentiated into neurons (using an inducible Neurogenin 2, Ngn2, cassette) and enable CRISPR inhibition gene knockdown (via stable expression of dCas9-BFP-KRAB). The combination of these elements in a cell line enables multiple lines of research, including small molecule screens for drug development in neuronal disease models, as well as studying stem cell biology in an iPSC neuronal cell model.

This technology includes an automated and non-invasive assessment system called Automated Identification of subjects at risks of Multiple Sclerosis (AIMS) to assist clinicians in their diagnostic evaluation. A focus of AIMS is the ‘central vein sign’ (and other radiological findings).

This technology provides a novel method of treating or preventing pruritis (itch) using natriuretic polypeptide b (Nppb) blocking agents. Itch (also known as pruritis) is a sensation that may be perceived as an unpleasant skin irritation and may drive an urge to scratch. Conditions such as, for example, psoriasis, atopic dermatitis, renal failure, liver cirrhosis and some cancers may cause persistent itch. Itch is triggered by somatosensory neurons expressing the ion channel TRPV1 (transient receptor potential cation channel subfamily V member 1).