Award Data

AbstractThe current estimate of the number of human genes is aboutin contrast to the number of presumed proteinswhich likely exceeds one millionThis increased complexity is the result of post transcriptional and post translational modificationsPTMsoccurring at multiple sites and in a quantitative variable mannerThe overwhelming result is a combinatorially expanding complexity of proteoforms that i ...

Project Summary While nucleic acid therapeutics have shown promise in rodent and nonhuman primate models of CNS diseasesall double blind clinical trials to date have failedOne likely explanation for this failure is poor therapeutic distribution in targeted brain regionsFor examplepost mortem studies of patients with Parkinsonandapos s Disease receiving intraparenchymal injections of AAV NTN demons ...

Neurodevelopmental diseasesNDDsAutism Spectrum DisorderASDIntellectual DisabilitiesBipolar disordersand Schizophreniaetcare prevalent and due to errors in neurodifferentiation and neurocircuitryWhile NDDsin many casesare due to inherited mutationsevidence is mounting that prenatal exposure to environmental toxicants also cause NDDs via disruption of epigeneticse gmodifications of histoneshistone t ...

PreeclampsiaPEis a pregnancy specific disorder characterized by hypertension and commonly by the appearance of urinary proteinThis disorder occurs inof pregnancies worldwide and is the second leading cause of maternal fetal morbidity and mortality worldwideIn low resource settingsPE accounts for up toof maternal deathsThe etiology of this disease is poorly understoodThis confounds therapeutic inte ...

PROJECT SUMMARY This proposal aims to develop a novel caveolae targeting antibody for rapid clinical translationIts long term objective is to develop the caveolae pumping systeman active transendothelial transport pathwayto provide an effective solution to the delivery and toxicity problem of systemically administered chemotoxinsWork will be based on the current proprietary mouse monoclonal antibo ...

New pharmacological treatments for epilepsy will be discovered through the development of new screening platforms that are rapidreliableand can identify novel disease modifying therapiesor anti epileptogenic treatmentsXona MicrofluidicLLCXonais in a unique position to provide such screening tools and services for epilepsy researchXona focuses on the development of microfluidic platforms for neuron ...

PROJECT SUMMARY ABSTRACT PEEL TherapeuticsIncis a life sciences company created to impact the lives of people with cancerBased on the research efforts of DrJoshua Schiffman and DrAvi SchroederPEEL TherapeuticsPEELhas created liposomal nanoparticles that have been loaded with elephant pEPproteins as a new class of anti cancer protein therapeuticsElephants have an unexpectedly low rate of cancer giv ...

PROJECT SUMMARY Modern DNA sequencing technologies have transformed our ability to interrogate human genomes in a single experimentthereby eliminating the inherent blind spots of gene panels and whole exome sequencingFurthermorerecent speed and economy improvements are driving the cost of whole genome sequencingWGSdown to that of WESthereforewe foresee a transition over the next two years to WGS a ...

PROJECT SUMMARYTraumatic Brain InjuryTBIis one of the most significant Public Health trauma diseases of our timeDiagnoses of various TBI forms rely on patient self reportingestablished clinical rating scalesand multimodality imaging to an extentTBI elicits complexheterogeneous pathological eventsincluding mechanical injuryhemorrhageand progressive secondary processes such as central and peripheral ...

Project SummaryNuclear magnetic resonanceNMRis amongst the most powerful analytical techniques ever inventedas recognized byNobel Prizes for methods development aloneNonethelessNMR is notoriously plagued by poor sensitivity and spatial resolutionState of the art NMR spectrometers typically feature detection thresholds ofnanograms forL sample volumesThis places NMR sensitivity many orders of magnit ...