Award Data

DESCRIPTION provided by applicant Adeno associated virus AAV is a non pathogenic human parvovirus and recombinant AAV vectors have gained significant attention owing to their safety and in some cases clinical efficacy in a number of Phase I II clinical trials However relatively large vector doses are needed to achieve clinical efficacy and such high doses can lead to host immune respon ...

PROJECT SUMMARY Thiopurines such as mercaptopurine are S-substituted antimetabolites used as functional analogs to natural nucleobase precursors. They are highly effective against hematologic malignancies (leukemia and lymphoma) and autoimmune diseases (inflammatory bowel diseases [IBD]), with an estimated patient population andgt;350,000 in the US. However, the cytotoxicity of thiopurines depends ...

PROJECT SUMMARY/ABSTRACT Opioid Use Disorder (OUD) is a chronic, recurring condition that if left untreated, poses a significant and ongoing public health threat. OUD is associated with significant impairments in physical and psychological health including increased mortality and high utilization of emergency medical care as well as increased risk of Hepatitis C and HIV infection and Neonatal Abst ...

Project Abstract An estimated 5.7 million Americans have Alzheimer’s disease or a related dementia (ADRD) and one in ten people age 65 or older show symptoms of Alzheimer’s disease. As ADRD progresses so does the level of dependency on others. As the disease progresses, higher levels of dependence in people with Alzheimer’s disease are associated with significantly increased burden and costs ...

ABSTRACT: Cutaneous vascular malformations (CVM), such as port wine stain (PWS) which occurs in 0.3–0.5% of newborns, are often a cause of great concern to patients for both medical and cosmetic reasons. Laser irradiation with flashlamp-pumped pulsed dye lasers, i.e. photothermolysis therapy (PTT), is currently the gold standard treatment for PWS. Most patients with PWS, however, fail to clear c ...

PROJECT SUMMARY Understanding of tumor molecular characteristics during a course of radiotherapy (RT) treatment is crucial to assess the efficacy of the treatment and adapt radiation dosage to optimize outcomes. Unfortunately, this cannot be routinely achieved by current diagnostic nuclear imaging modalities due to complex logistics and prohibitive costs. Alternatively, Cerenkov Emission (CE) is a ...

Abstract Aggregation of mutated myocilin (MYOC), a protein found in the endoplasmic reticulum (ER) of trabecular meshwork (TM) cells, disrupts the outflow of aqueous humor from the eye, causing increase in intraocular pressure (IOP), progressive retinal ganglion cell (RGC) death, and degeneration of the optic nerve, leading to primary open-angle glaucoma (POAG), characterized by irreversible visio ...

PROJECT SUMMARY FOR PREDICATE STTR PHASE I GRANT Perioperative and prescribed opioids often result in costly and unpredictable adverse effects, including life threatening respiratory depression and long-term opioid use/misuse in vulnerable patients. The US FDA warns against the use of codeine and tramadol in children due to postoperative anoxic brain injuries and deaths, and in nursing mothers due ...

Mekanistic Therapeutics seeks to design, discover, and develop anti-cancer agents that selectively inhibit multiple oncogenic pathways. Among Mekanistic’s portfolio are dual and highly selective inhibitors of EGFR and PI3 kinase, which were rationally designed to only target these two critical oncogenes. The PI3 kinase (PI3K)/AKT/mTOR pathway plays a central role in driving tumor cell survival a ...

ABSTRACT Many clinical situations, including stroke, expose the brain to insufficient cerebral blood flow (CBF) that cannot maintain normal cerebral metabolic rate of oxygen consumption (CMRO2) requirements, thereby leading to cerebral ischemic/hypoxic stresses and neurological disorders. Effective interventions are dependent on the findings of CBF/CMRO2 improvement and eventually neural recovery. ...