Award Data

ABSTRACT Advances in molecular biology and genetic engineering have led to the design and use of modified T cells recognize tumors to achieve significant tumor control upon adoptive cell transfer (ACT) to patients. These T cells are either transduced with tumor antigen reactive T cell receptors (TCR), or chimeric antigen receptors (CARs). Recently, a surge in studies with neo-antigen reactive T ce ...

PROJECT SUMMARYRNA is a validated target for drug design, both as therapeutic and as a target. Targeting specific RNA, such as rRNA which are involved in proliferation and survival of bacteria is a promising approach. We are developing fast and low cost methods to screen sequence-specific small molecules for novel anti-ribosomal activities. We will construct sequence-specific chemically modified r ...

Project Summary Proliferative vitreoretinopathy (PVR) is a major complication of retinal detachments and is responsible for most of the failures in retinal reattachment surgeries. About 10% of retinal detachment patients suffer from PVR. These patients often have poor visual outcome despite multiple surgeries. Our previous work on inhibition of PVR has focused on pharmacological intervention concu ...

ABSTRACT This Phase STTR Phase I proposal aims to engineer a synthetic metabolic pathway in a microbial host to produce UDCA and related compounds. The proposed work has high intellectual merit for the following reasons: (i) UDCA biosynthesis does not occur in any organism whose cultivation can be scaled to meet global demand, (ii) UDCA requires a multi-organism biosynthetic pathway (animal cholic ...

ABSTRACT Glaucoma is the leading cause of irreversible blindness in the world. The number of people with primary open angle glaucoma is expected to exceed 100 million by 2040. The first-line therapy for glaucoma treatment is patient-administered hypotensive medication delivered topically via an eye drop. However, studies have consistently shown that patients rarely adhere to dosing recommendations ...

Abstract Duchenne Muscular Dystrophy (DMD) is a fatal muscle disease with a predicted incidence of 1 in 5000 males. DMD results from mutations in the gene encoding dystrophin, a 427 kDa scaffolding protein responsible for providing a mechanical link between the muscle fiber actin cytoskeleton and laminin in the extracellular matrix. The 7β1 integrin is a transmembrane linkage system in skeleta ...

PROJECT SUMMARY . Collecting complete and accurate outcome data directly from research participants is becoming increasingly important. Clinical researchers needs a cost-effective approach to capture high-quality patient-reported outcomes. Typically, data captured directly from participants is through self-administered questionnaires or through a human interviewer, each with their own advantages ...

PROJECT SUMMARY The long-term goal of the current proposal is to generate a new class of small molecules to treat familial hypercholesterolemia. Familial hypercholesterolemia (FH) patients suffer from excessively high levels of Low Density Lipoprotein Cholesterol (LDL-C), which if left untreated results in death from severe cardiovascular disease. FH is caused primarily by mutations in the LDL rec ...

Abstract Building on our successful primate model studies, we aim to develop a one-time treatment for durable remission of human immunodeficiency virus (HIV) in the absence of antiretroviral medications. This treatment is an autologous HIV-specific chimeric antigen receptor (CAR, specifically CD4-MBL-CAR) T cell therapy that targets B cell follicles1,2. B cell follicles are an immune protected sit ...

PROJECT SUMMARYCarotid Artery Stenting (CAS) using embolic protection represents the current standard of care for patients with carotid artery stenosis considered at high risk for carotid endarterectomy. Distal filter devices are the most widely used protection devices, but these must be navigated through plaque prior to deployment, presenting a significant embolic risk. Strong evidence indicates ...