Award Data

Cryptococcus species are a clinically important group of opportunistic fungal pathogens that can cause life threatening disease, particularly in the immunocompromised patient population. Historically, this has involved patients with advanced HIV, but is becoming more prevalent in other immunovulnerable populations. The primary pathogens, C. neoformans and C. gattii, are ubiquitous in the environme ...

PROJECT SUMMARY The epidermal growth factor receptor (EGFR) gene is mutated and/or amplified in majority of primary glioblastoma (GBM). While EGFR-mutant GBM cancer cells are dependent on EGFR signaling for survival, numerous small molecule EGFR tyrosine kinase inhibitors (TKIs) have failed to show efficacy in this disease. There are two main reasons for these failures: i) Many of these EGFR-TKIs ...

PROJECT SUMMARY Despite the enormous value of radiopharmaceuticals for imaging and therapy in clinical nuclear medicine, and for research and drug development, the production of these compounds remains very expensive because of the need for complex, capital-intensive equipment and infrastructure (i.e., hot cells, stack monitor, radiosynthesizer, and a suite of QC testing equipment) at each site. F ...

Summary/abstract. Leptospirosis is a globally important neglected disease caused by pathogenic Leptospira. It is estimated to cause more than 1 million global cases annually with a 5-20% case-fatality rate, significant morbidity, and important public health consequences. Currently there is no safe and effective vaccine to prevent human leptospirosis. LeptoX, Inc. proposes to develop the first huma ...

Abstract The Specific Aim of this Phase I TTR proposal is to test the feasibility of our siRNA drug to ameliorate the pathology and progression of alcohol-associated liver disease (ALD). Alcohol consumption remains a leading cause of hepatic pathology worldwide and is one of the greatest sources of preventable morbidity and mortality. In the U.S., alcohol abuse impacts over 10 million individuals ...

Abstract Sepsis, a heterogeneous syndrome characterized by whole-body inflammation caused by the body's response to an infection, is the most expensive and deadly condition treated in hospitals, with over 270,000 cases of sepsis-related deaths in the U.S. alone. The cornerstones of optimal sepsis care are early recognition accompanied by appropriate antimicrobial therapy, and use of evidence-based ...

Summary Mild cognitive impairment (MCI) represents an intermediate state between normal aging and the diagnosis of clinically probable very early Alzheimer's disease (AD). Since MCI manifests before the onset of AD, it may be the optimal stage in which to intervene with preventative therapies for AD and age-related dementia before progressive neurological loss and irreversible cognitive impairment ...

PROJECT ABSTRACT The goal of this Phase I STTR project is to accomplish key milestones in commercializing a novel SaCas9 nickase-based system (CRD-002) to treat dystrophinopathies in order to restore the production of healthy, endogenous full-length dystrophin protein otherwise compromised by duplication mutations. Duchenne muscular dystrophy (DMD) is characterized by progressive muscle loss and c ...

PROJECT SUMMARY 5-ALA-induced PpIX fluorescence-guided surgery (FGS) enables real-time visualization of residual cancer tissue during resection of brain tumors. This addresses a critical need in surgical oncology as the ability to identify residual tumor leads to improvements in overall survival, whereas the sparing of normal tissue benefits the patient’s quality of life. Currently, surgeons vis ...

1 Approximately 6.5 million Americans aged 65 and older live with dementia, supported by more than 11 million2 unpaid family caregivers. These numbers are projected to rise exponentially in the coming decades fueling the3 need to develop scalable ways to support dementia caregivers. The need to empower dementia caregivers to4 continue providing care while maintaining their own health and well-bein ...