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DESCRIPTION provided by applicant Phenomenal advances in DNA sequencing technologies have enabled systematic identification of genetic variants in human individuals and the recent FDA marketing authorization of the first next generation genome sequencer signals the arrival of a new era of pharmacogenomics and personalized medicine Nevertheless DNA sequencing alone fails to provide complete i ...

Project Summary Traumatic brain injuryTBIcauses detrimental behavioral dysfunctions and brain neurodegeneration butunfortunatelythere currently is no effective pharmaceutical TBI treatmentThe underlying PhaseSTTR grant is to develop a new class of TBI drugs that inhibit the protease cathepsin BGenetically deleting the cathepsin B gene in mice results in substantial behavioral and pathology improve ...

DESCRIPTION provided by applicant There are approximately million cases of dengue fever each year with billion people at risk mostly in low income countries There are four serotypes of dengue fever virus DENV and in its uncomplicated form the disease is worthy of its indigenous name andquot break bone feverandquot An individual may suffer sequential infections with multiple DE ...

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 ...

ABSTRACT Stroke is a leading cause of disability worldwideGloballythere aremillion stroke survivors each year who have significant neurological deficits including sensory and motor disabilityleading to excessive socioeconomic burdenCell therapy using primary MSCs has been actively explored by us and others as a therapeutic solution to this unmet medical needHoweversince the therapeutic efficacy of ...

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 ...

DESCRIPTION provided by applicant The knockdown of targeted genes by anti sense oligonucleotides ODNS and genetic medicines collectively G MEDS holds promise for a variety of therapies The delivery of effective quantities of ODNS to specific cells however has proved to be challenging We propose here a novel approach to ODN delivery that involves enveloped virus like particles EVLPs ...

DESCRIPTION provided by applicant Activation of lung myofibroblasts LMF is responsible for the development of lung fibrosis in chronic lung diseases of all causes and remarkably LMF clearance by apoptosis may prevent development of lung fibrosis and lung injury and possibly allow recovery from reversal of lung fibrosis There is full agreement among tissue fibrosis experts that inhibiting o ...

Project Summary Abstract Many pharmaceutical agents are highly potentbut are unable to exert substantial therapeutic activity against disorders of the brainbecause the blood brain barrierBBBeffectively prevents their access to the site of intracerebral diseaseFor examplethe majority of glioblastoma patientsdespite the best efforts of current medical caredie within two years after diagnosisHencethe ...