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 SUMMARY ABSTRACT Interactions between the gut microbiome and the immune system have been linked to inflammatory bowel disease IBD IBD is a chronic and progressive gastrointestinal disease characterized by uncontrolled activation of the intestinal immune system resulting in severe medical complications affecting over million patients in the United States It is a disease of high u ...

Summary In this fast track Phase I II STTR project a novel mass spectrometry method and a matched reagent kit will be developed for mapping modified nucleotides in human mitochondrial transfer RNA mt tRNA including the most abundant modification pseudouridine which hitherto has proved to be a technical challenge because it is an isomer of uridine These new research tools will be harnessed to build ...

Project Summary Linking infectious agent diagnostics to clinical decision making at the point of care requires a fast highly sensitive and simple to use method that takes into account the infrastructure and workflow in clinical settings of both developing and developed regions In addition being able to distinguish among multiple potential infectious pathogens in a single test is of great impor ...

DESCRIPTION provided by applicant There are currently no drugs or other therapeutic interventions that can reverse or halt the progression of Alzheimerandapos s disease AD Age is by far the greatest risk factor for AD and it is known from studies in both mice and humans that neuroinflammation is increased with old age and to an even greater extent in AD Therefore a drug that could reduce ...

DESCRIPTIONprovided by applicantThe purpose of this project is to develop an improvedminimally invasive treatment for intracranial aneurysmsIntracranial aneurysms are spherical outpouchings of blood vessels in the head that result from weakness in the vessel wallUnruptured aneurysms are present in approximatelyof the general populationand rupture can be devastating with a high morbidity and mortal ...

DESCRIPTION provided by applicant Genomic instability is a hallmark of cancer and represents a targetable vulnerability yet is underdeveloped as a therapeutic area The central goal of this Phase program is to complete the early preclinical development of a new class of new cancer therapeutics that uniquely target RAD delivering effective cancer cell selective therapy in subsets of bioma ...

DESCRIPTION provided by applicant Gene transfer for the treatment of cardiovascular diseases is bedeviled by inability to obtain safely and easily sufficient cardiac transgene expression Current methods of gene transfer for heart disease include intramuscular injection into heart muscle or intracoronary delivery approaches that are cumbersome to apply Consequently we have considered the u ...

DESCRIPTION provided by applicant Atopic dermatitis AD or severe eczema is a very common life long skin disease with of adults in the US requiring systemic therapy Clinical manifestations of AD include multiple inflamed lesions erosions accompanied by lichenification thick leathery skin fibrotic papules and severely dry skin with increased susceptibility to infection A major uncont ...

DESCRIPTIONprovided by applicantThe goal of this Fast Track STTR project is to develop a novel CardioPortTM surgical device that will enable minimally invasive beating heart repair without use of cardiopulmonary bypassCPBThere is a critical unmet need for new instruments to improve the surgical repair of heart defects in childrenCurrent best surgical practices involve either open heart surgery wit ...