Award Data

PROJECT SUMMARY/ABSTRACT In response to National Institute on Aging's (NIA) strategic plan and PAS-19-317, we propose novel therapeuticsto treat Alzheimer disease and related dementias (ADRDs). We have developed novel combinations of three natural product compounds, which modulate complementary pathways in multiple types of CNS cells (i.e., neurons, microglia) associated with AD pathology. These c ...

PROJECT SUMMARY Actively involving informal caregivers in direct patient care at the bedside in the intensive care unit (ICU) has the potential to improve patient, informal caregiver, and health care system outcomes. Practice guidelines call for the liberal inclusion of informal caregivers as active partners in ICU care, but there is a critical, unmet need to develop tools that effectively operati ...

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 ...

Abstract Currently, clinically used drug abuse medications exist for treatment of addiction to opiates, alcohol, and nicotine, but not psychostimulants, such as methamphetamine (METH). Compounds that co-activate both nociceptin opioid peptide (NOP) and mu receptors have potential for treatment of drug abuse. In particular, buprenorphine, a partial mu agonist/kappa antagonist, which also acts as a ...

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 ...

Project Summary The proposed project will develop a breakthrough endovascular sensor technology to allow for real-time quantification of the mitral coaptation force (MCF) of the mitral valve during both open and percutaneous mitral valve repair (MVR). Mitral regurgitation (MR) is the most common heart valve disorder in the U.S. population, with over 2 million individuals affected. Current methods ...

ABSTRACT Suicide is a leading cause of death in the US and suicide rates have steadily increased. Despite this, there is a paucity of interventions that reduce suicide ideation and behavior. In this project, we seek to enhance a beta version of our suicide prevention digital therapeutic (developed in phase I) and conduct a randomized controlled trial to evaluate the efficacy of the digital therape ...

PROJECT SUMMARY Data is a critical and highly valuable commodity, driving meaningful change in our society, especially when it pertains to patient care and biomedical research. Currently, institutions pay inordinate sums to increase, regain, and complement their data panels. As an extra burden, data legislation and privacy protection regulations introduce barriers to forming effective partnerships ...

ABSTRACT SaCas9 is a major gene editing nuclease that is preferred for in vivo applications thanks to its relatively smaller size compared to that of spCas9. One limiting factor for the use of saCas9 is its strict PAM requirement of NNGRRT. In addition, there are limited efforts to reduce saCas9’s off-target editing rates. In Aim 1, we propose a computational approach to relax saCas9’s PAM req ...