Award Data

PROJECT SUMMARY Congenital Myasthenic Syndromes (CMS) are a group of clinically similar neuromuscular transmission disorders that differ in their underlying genetic mutation. While some phenotypical variation exists, CMS are commonly characterized by muscle weakness (myasthenia) that worsens with physical exertion. Defects in Collagen Q (ColQ), a multidomain functional protein of the Neuro Muscula ...

For more than a decade, our work has focused on development of therapeutic interventions for viral- and bacterial-induced acute lung injury (ALI) and the more severe acute respiratory distress syndrome (ARDS). Significantly, we identified the Toll-like receptor 4 (TLR4) signaling pathway as key to the host response to influenza and secondary bacterial infection following influenza. We also identif ...

Project Summary There are 24,000 stillbirths per year in the United States, and 2.6 million stillbirths per year worldwide, with no significant decrease in the last decade. Abnormal fetal heart rhythms, such as long QT syndrome, are thought to play an important role in these deaths, and may cause 3- 10% of unexplained stillbirths. This is in addition to known fetal arrhythmias, which affect 1-3% o ...

Abstract: Non-alcoholic fatty liver disease (NAFLD) is one of the most common liver diseases in children and adults associated with diet-associated hepatic lipid accumulation (steatosis), and is a common hepatic manifestation of obesity and metabolic syndromes including diabetes. Modulation Therapeutics is developing a stearoyl- coenzyme A desaturase-1 (SCD1) inhibitor for the treatment of NAFLD a ...

Project Summary/Abstract for STTR Phase I Application: An immunotherapeutic IgY formulation against norovirus diarrhea Human norovirus (HuNoV) infects millions of people globally each year, causing diarrheal illness resulting in more than 200,000 deaths annually, particularly in the very young, the very old, and the immunocompromised. There is currently neither an effective vaccine nor effective t ...

ABSTRACTGlobally, more than 71 million people are infected with Hepatitis C virus (HCV), with 1-2 million new infections occurring each year. This major health concern necessitates the development of an effective vaccine. Since HCV rapidly accumulates mutations, vaccines must elicit the production of broadly neutralizing antibodies (bnAbs) in a reproducible fashion. The viral envelope E1E2 glycopr ...

PROJECT SUMMARY Microinjection technologies underlie many research and clinical applications that require gene and cell delivery, including studies and emerging treatments of neurological conditions (e.g., neurodegenerative diseases, traumatic brain injury, and cancer). Unfortunately, challenges associated with the microinjection tools by which the viral vectors and cells inherent in these applica ...

Abstract: Developing a Digital Training and Assessment Platform to Disseminate a Successful NIH- Supported College Preparation Model for Diversifying the U.S. Biomedical Workforce This Phase 1 SBIR project will design and test a digital training and assessment platform—HSTA Hatch—to help geographic regions outside West Virginia (WV) establish workforce development programs for grade 9-12 youth ...

Abstract Over 5,000 lung transplants (LTx) are performed in the US each year to save the lives of patients in respiratory failure due to COPD, idiopathic pulmonary fibrosis, cystic fibrosis, pulmonary hypertension, and other terminal lung conditions. The vast majority of LTx, long-term survival is significantly limited by chronic lung allograft dysfunction (CLAD) with a median survival of 6 years ...

Project Abstract In 2020 malaria cases (241M) and deaths (627,000) reached the highest since 2012 emphasizing the urgent need for new tools for prevention, control, and elimination of this disease. Sanaria’s Plasmodium falciparum (Pf) sporozoite (SPZ) Vaccine, composed of radiation attenuated PfSPZ administered by intravenous injection, assessed in 1740 subjects aged 5 months to 61 years in 6 co ...