Award Data

ABSTRACT SUMMARY Pyruvate dehydrogenase complex PDC deficiency PDCD is a rare disease of mitochondrial energy failure in which the life of expectancy of affected children is severely truncated Treatment of PDCD remains a serious unmet challenge There has never been a controlled trial of any intervention for PDCD thus there is no proven therapy for affected patients Dichloroacetate DCA ...

Project Abstract/Summary Pyruvate dehydrogenase complex (PDC) deficiency (PDCD) is a rare disease of mitochondrial energy failure in which the life expectancy of affected children is severely truncated from unrelenting lactic acidosis and/or from progressive neurological and neuromuscular degeneration. Treatment of PDCD remains a serious, unmet, challenge. Dichloroacetate (DCA) represents the firs ...

DESCRIPTION provided by applicant Fibroproliferative illnesses leading to organ fibrosis and failure are responsible for approximately of deaths in developed countries whether idiopathic triggered by environmental factors infections or genetics organ fibrosis results in significant morbidity and mortality Organ fibrosis is responsible for health care costs exceeding $ billion year ...

DESCRIPTION provided by applicant Fibroproliferative illnesses leading to organ fibrosis and failure are responsible for approximately of deaths in developed countries whether idiopathic triggered by environmental factors infections or genetics organ fibrosis results in significant morbidity and mortality Organ fibrosis is responsible for health care costs exceeding $ billion year ...

DESCRIPTION provided by applicant Dental caries is the single most common affliction across the globe affecting billion people The current process of identifying dental caries involves examining caries formation by naked eyes or X ray during routine checkups Once identified surgical restorative procedures are used to drill out the decay portion of the tooth an filled with synthetic mate ...

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 ...

Neurodon LLC proposes to conduct lead optimization and candidate-seeking activities on a novel series of neuroprotective small molecules that shows efficacy in a transgenic model of Alzheimer’s disease (AD). AD is a leading cause of death in the United States, with some estimates ranking it as high as third behind cardiovascular disease and cancer. Despite the enormity of this national public he ...

This grant is for translational development of a scalable, nucleic acid-based formulation of cytomegalovirus-vectored vaccines that can be distributed without a cold chain. HIV candidate vaccines that use cytomegalovirus (CMV) as delivery vector and immunomodulatory adjuvant have shown extraordinary promise. In our preliminary work, for example, a rhesus cytomegalovirus-vectored SIV vaccine lackin ...

DESCRIPTION provided by applicant The pursuit of novel therapies informed by the astounding advances in biomedical science of the past half century has been fruitful for many disorders However inherited muscle diseases such as muscular dystrophy remain stubbornly resistant to sophisticated molecular approaches to therapy A few therapies are finally edging towards approval but it has become ...

DESCRIPTION provided by applicant Most samples of chemical interest are complex mixtures making the usual combination of chromatography with mass spectrometry MS a natural choice However the increased demand for chemical analysis by mass spectrometry in many areas of science makes it imperative to increase efficiency of analysis by minimizing sample workup and overall analysis time which ...