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Summary The goal of this study is to discover novelmechanism based pharmacological intervention for autisma devastating neurodevelopmental disorder with no treatment currentlyGenetic sequencing has revealed extensive overlap in risk genes for autism and for cancermany of which are chromatin remodeling factors important for transcriptional regulationsuggesting the possibility of repurposing the ant ...

Project SummaryA Novel Polymeric Valve for Transcatheter Aortic Valve Replacement Minimally invasive transcatheter aortic valve replacementTAVRhas emerged as an effective therapy for the unmet clinical need of inoperable patients with severe aortic stenosisASRecent longitudinal follow up studies of TAVR patients however indicate that this procedure and associated technology may result in serious a ...

PROJECT DESCRIPTIONThis STTR Phase I proposal is intended to demonstrate the feasibility of developing tanshinone small molecule analogTSAas an effective and safe small molecule therapy for patients with sepsis and septic shockIn the USmore than one million people develop sepsis every yearYetno effective anti sepsis treatment exists other than antibiotics and supportive careWe have discovered that ...

Ultrasound Based Device to Guide Treatment of Graft Versus Host Disease using Skin Elasticity as a Biomarker ABSTRACT Sclerotic chronic graft versus host disease cGVHD develops in of allogenic Human Stem Cell Transplant HCT recipients and is associated with significant morbidity and mortality GVHD is treated with immunosuppression which puts patients at severe risk of infection ...

PROJECT SUMMARY Dermal scarring affects more than million people worldwide annually over million people are injured in motor vehicle accidents over million patients are severely burned and thousands of warriors are wounded in military blasts In severe burns more than of patients develop hypertrophic scar contraction which leads to hypertrophic scar contractures HSc HSc a ...

Project Summary This study aims to prove the concept and feasibility that TendonCure extracellular vesicles that contain signaling molecules i e exosomes secreted by mesenchymal stem cells i e tendon stem progenitor cells TSPCs or adipose derived stem cells ADSCs from individual patients donors cultured on a novel scaffold exert therapeutic efficacy on tendinopathy when injected into ...

ABSTRACT Alzheimerandapos s and other tauopathies are medical problems growing to historical proportions that threaten the long term viability of medical care systems world wide Alzheimerandapos s costs today are of the US GDP and growing as the population ages Effective disease modifying treatments have yet to be developed A number of drugs in clinical testing target amyloid but very fe ...

Mitochondria are the principal generators of cellular ATP by oxidative phosphorylation which links electron transport via Complexes I through IV to production of ATP via ATP synthase Over pathogenic human mitochondrial DNA mtDNA mutations associated with diseases ranging from cardiomyopathy to juvenile blindness have been identified Collectively millions are affected by mtDNA disease fo ...

Project titleDeveloping heparan sulfate glycan array This proposal is aimed at developing a high throughput method to study the interactions between heparan sulfateHSand proteinsHS is a highly sulfated polysaccharide displaying a wide range of biological functionsGlycan Therapeutics is a company specialized in HS related research productsIn this proposalan HS array technique will be constructed us ...

PROJECT SUMMARY Lysosomal storage diseases LSD are rare inherited metabolic disorders caused by defects in the cellular catabolic system Mucopolysaccharidosis Type IIIC MPS IIIC or Sanfilippo disease type C is one such LSD that is caused by deficiency of the enzyme heparan sulfate acetyl CoA glucosaminide N acetyltransferase HGSNAT essential for degradation of heparan sulfate a repeati ...