Award Data

DESCRIPTION (provided by applicant): Mucopolysaccharidosis type I (MPS I) is an autosomal recessive storage disease caused by the absence of a-L-iduronidase (IDUA), resulting in systemic accumulation of glycosaminoglycan (GAG) storage materials, hepatosplenomegaly, skeletal dysplasias, cardiopulmonary obstruction, progressive neurologic impairment and death by age 15. MPS I is currently treated b ...

DESCRIPTION (provided by applicant): Approximately 30,000 people in the United States are diagnosed each year with pancreatic Cancer (PanCa). Over 90% of these will die of the disease making PanCa one of the most devastating human malignancies. The current 5-year survival rate of less than 4% has changed little in the last 20 years despite improvements in therapy including the introduction of chem ...

DESCRIPTION (provided by applicant): Approximately 30,000 people in the United States are diagnosed each year with pancreatic cancer (PanCa). Over 90% of these will die of the disease making PanCa one of the most devastating human malignancies. The current 5-year survival rate of less than 4% has changed little in the last 20 years despite improvements in therapy including the introduction of ...

DESCRIPTION (Applicant's Abstract): The investigators have developed a plasmid vector that expresses the normal human alpha-1 antitrypsin (AAT) gene in human respiratory epithelium when delivered as a plasmid-cationic liposome complex. In subjects with inherited AAT deficiency, they showed that liposome mediated transfer of the normal AAT gene to the nasal respiratory epi ...

DESCRIPTION (provided by applicant): Molecular interactions are central to the understanding of basic biology and cellular function, are paramount to all molecular assays (research and in vitro diagnostics), are essential in biomarker discovery and critical toward evaluating therapeutics. However, the tools available to quantify these interactions have limitations, in so far as they either rely up ...

DESCRIPTION (provided by applicant): Molecular interactions form the foundation of biology and chemistry. They are central to life itself and determine catalytic activity, cellular function, and therapeutic efficacy. The vast majority of diagnostic procedures depend on some type of specific molecular interaction. Therefore, the ability to perform pure liquid-phase molecular binding analysis at hi ...

DESCRIPTION (provided by applicant): The long term objective of this proposal is to develop a diagnostic kit for monitoring nonenzymatically galactated proteins in galactose. Classical galactose is a disorder of galactose metabolism caused by a deficiency of galactose-1-phosphate uridyltransferase. The resulting severe impairment of galactose metabolism has been recognized for half a century and t ...

PROJECT SUMMARY Drug overdoses are the leading cause of death in the United States with approximately 72,000 people dying from drug overdose in 2017. Many of these fatalities occurred in individuals with opioid use disorder (OUD), which is one of the greatest public health issues confronting the United States today. Digital Recovery Support Services (DRSs) that include peer support, psychoeducatio ...

DESCRIPTION (provided by applicant): Atherosclerosis is the underlying cause of most cardiovascular disease and considerable evidence supports the role of hypercholesterolemia as a risk factor for this disease. In preliminary work, we found that tertiary-butyl hydroquinone (TBHQ), at a dose lower than approved for human use, was effective in reducing plasma levels of both total cholesterol and tri ...

DESCRIPTION provided by applicant Problem Lower gastrointestinal bleeding is a significant medical problem in the United States accounting for more than hospital admissions and tens of billions of dollars of healthcare spending annually It is common for bleeding from a single site to stop and restart multiple times The three modalities now used to diagnose and locate gastrointestina ...