Award Data

The lack of patient reported outcome measures (PROMs) appropriate for youth with neurodevelopmental disabilities (DD) and related cognitive impairments including Down syndrome (DS) poses a significant problem for healthcare research and practice. Parents and other professionals do not identify the same needs as youth, and failure to engage youth with DD in healthcare evaluation compromises healthc ...

Contact PD/PI: GILGER, BRIAN C Abstract Corneal blindness is a leading cause of global blindness, with allogeneic corneal transplantation (CT) being the most common form of tissue transplantation worldwide. Though approximately 180,000 CT surgeries are carried out each year, around 12.7 million are currently awaiting a donor cornea to undergo the operation. Still, CT surgery is accompanied by a hi ...

PROJECT SUMMARYSepsis is a life-threatening syndrome that involves a systemic inflammatory response that is initiated by an infection. There are an estimated 1.5 million of cases annually in the US with 250,000 resulting in death. The number of cases and associated deaths is expected to increase given the rising at-risk population of elderly individuals and the compounding challenge of antibiotic ...

PROJECT SUMMARY/ABSTRACT Autonomic dysreflexia (AD) is a potentially life-threatening hypertensive crisis that predominantly affects individuals with a spinal cord injury (SCI) above T6. AD can be triggered idiopathically or by distention or manipulation of pelvic visceral organs, especially the bladder and bowel, which can occur with catheterization and fecal evacuation procedures that are necess ...

Project Summary Natural products have served as a productive source of drugs over the past decades and have represented, or been the inspiration for, a majority of antimicrobial compounds that have entered the clinic. The goal of this proposal is to develop novel antibacterial adjuvants based on the marine natural product synoxazolidinone B to treat Gram-positive orthopedic joint infections. These ...

We recently developed a technology that combines catalytically inactive dCas9 with bifunctional chemicals that link endogenous host cellular machinery to specific genes targeted with dCas9. We have demonstrated this technology can significantly increase gene expression at multiple endogenous mammalian genes in a chemically dependent manner. Here we propose to apply this technology to the mono-alle ...

1 ABSTRACT2 The temporomandibular joint (TMJ) is a complex joint system critical for dental occlusion, mastication, 3 respiration and speech. The TMJ is comprised of a network of muscles, ligaments, and a4 fibrocartilaginous disc and condyle. Temporomandibular disorders (TMDs) afflict over 10 million5 Americans at an annual cost of ~$4 billion, per the NIDCR. Degenerative types of TMDs, including6 ...

Abstract. In partnership with the Crystal laboratory, Weill Cornell, LEXEO is developing an adeno-associ- ated virus (AAV)-based gene therapy to treat the central nervous system (CNS) manifestations of CLN2 (Batten) disease, a fatal, childhood autosomal recessive neurodegenerative lysosomal storage disorder caused by mutations in the CLN2 gene, coding for a lysosomal enzyme, tripeptidyl peptidase ...

Abstract One of the most severe foodborne outbreaks in Europe, and the second-largest “Shiga toxin-producing” E. coli outbreak worldwide, was traced to E. coli O104: H4 in fresh fenugreek sprouts from a farm in Germany. It affected more than 4,075 individuals in 16 countries and included 908 cases complicated by hemolytic uremic syndrome (HUS) and 50 deaths. In the U.S., sprouts were implicate ...

Abstract. LEXEO Therapeutics, LLC, is an early stage biotechnology company focused on using in vivo gene therapy technologies to treat hereditary disorders of unmet medical need. LEXEO is developing an in vivo gene therapy strategy to mitigate the high risk for osteoporosis in individuals with aldehyde dehydro- genase 2 (ALDH2) deficiency, a hereditary disorder affecting 8% of the world population ...