Award Data

Project Summary Sanfilippo disease (mucopolysaccharidosis type III; MPS III) is a devastating neurodegenerative lysosomal storage disorder of childhood whose pathologic features are neurologic: slowing of development, severe behavioral problems, progressive cognitive decline, dementia, and decline in motor skills leading to immobility, unresponsiveness, and death. We have focused on MPS IIID cause ...

Motor development plays a pivotal role in an infant’s overall development and has a cascading effect on social, cognitive, memory, and language ability. These functions can be compromised when motor development is disrupted; motor disruption is associated with generalized developmental delays. Early screening and identification of delays can allow early interventions with the goal of not just im ...

The chemokine receptors CXCR1 and CXCR2 (CXCR1/2) are validated as having essential roles in the growth, survival, motility, invasion and angiogenesis of human melanoma, which secretes abundant amounts of the corresponding chemokine ligands, including CXCL8. Additionally, abnormal cancer-induced immunosuppressive myeloid-derived suppressor cells (MDSCs) in the circulation and tumor correlate with ...

Project Summary. The Indicator Cell Assay Platform (iCAP) is an inexpensive blood-based assay that can be used for early detection of disease, disease stage stratification, prognosis and response to therapy for a variety of diseases. The iCAP uses cultured, standardized cells as biosensors, capitalizing on the ability of cells to respond to disease signals present in serum with exquisite sensitivi ...

Photodynamic therapy (PDT) is a cancer treatment that utilizes visible or near-infrared light to activate a light- sensitive drug (photosensitizer, PS) that, in turn, creates reactive singlet oxygen species from ground triplet state oxygen that is present in the tumor. The resulting reactive products induce tissue death. Ideally the reactive species will be produced within only the target volume, ...

Packed red blood cellpRBCtransfusions save livesHoweverhundreds of thousands of non hemolytic transfusion reactions occur each year worldwideranging from mild fever and pruritisto serious lifethreatening reactions such as severe hyperkalemiaanaphylaxisand transfusion related acute lung injuryTRALIMild moderate reactions drive patient discomfortdocumentationand testing costs while serious reactions ...

7. Project Summary/Abstract: Outbreaks of infection caused by contaminated endoscopes are a serious public health problem that has even resulted in patient deaths. Currently, because of the narrowness of the channels and complexity of the endoscope, there simply is no reliable way of achieving this. The existing cleaning protocols also are highly dependent on operator technique. Accordingly, this ...

Project Summary Current SARS-CoV-2 molecular diagnosis tests mostly are based on real-time quantitative PCR (qPCR). Digital PCR is a next-generation PCR technology based on limiting dilution, end-point PCR, and Poisson statistics. Digital PCR transforms the exponential, analog nature of qPCR quantification into a linear, digital signal quantification. Compared to qPCR, digital PCR offers several i ...

ImmuNext has defined an anti-MCT1 monoclonal antibody (mAb) that will be developed for the treatment of human autoimmune disease. The membrane monocarboxylate nutrient transporter SLC16A1 (MCT1) is a multi-pass transmembrane protein responsible for the facilitated transport of critical metabolites, including products of glycolysis: lactate, pyruvate and ketones. Our strong preliminary data support ...

Packed red blood cell (pRBC) transfusions save lives. However, hundreds of thousands of non-hemolytic transfusion reactions occur each year worldwide, ranging from mild fever and pruritis, to serious life- threatening reactions such as severe hyperkalemia, anaphylaxis, and transfusion-related acute lung injury (TRALI). Mild-moderate reactions drive patient discomfort, documentation, and testing co ...