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AbstractLEXEO TherapeuticsLLCis an early stage biotechnology company focused on using gene therapy technologies to protect vulnerable organs from oxidant stressLEXthest LEXEO productis ast in classnext generation gene therapy treatment for alphaantitrypsinAATdeficiencyan autosomal recessive hereditary disorder associated with low serum levels of AATAATSERPINAakDa serine antiprotease produced by th ...

Project Summary As part of the FDA approval process for novel therapeutic drugssafety screening demonstrating that the candidate drug does not have deleterious effects on the human cardiac action potential is requiredHealthy human heart cells are not available for such preclinical drug testingThere is currently no consensus on the best experimental approach to provide the most efficienteconomicala ...

Project Summary Abstract Children with speech sound disorder show diminished accuracy and intelligibility in spoken communication and may thus be perceived as less capable or intelligent than peerseven when they are of average or above average intellectual abilityWhile most speech errors resolve by the late school age yearsup toof individuals will continue to exhibit residual speech errorsRSEthrou ...

AbstractA major shortfall in clinical care for diabetes is a rapid and accurate screen for progression to diseaseCurrent criteria involve HLA haplotypeand the presence of auto antibodiesAAGenome wide associated studiesGWASrevealed only immune associated genes including HLA as T D predictorsThe ability to differentiate from other diseases was very limitedPre typediabetesp T Dis now being identified ...

Psychostimulant abuse is a significant public health problemwithmillion Americans reporting cocaine use andmillion reporting methamphetamine useCurrentlythere are no FDA approved pharmacotherapies to treat stimulant dependencePrevious cocaine medications development efforts have focused on small molecule drugs as they can readily enter the brainThe lack of brain penetrance by large molecules was s ...

ABSTRACTPrematurity is the largest single cause of death in children under five in the world and lower respiratory tract infectionsLRTIare the top cause of hospitalization and mortality in premature infantsClinical tools to predict and prevent severe LRTI in premature pediatric patients are critically needed to allow early interventions to decrease the high morbidity and mortality in this patient ...

Abstract Atrial fibrillationAFprevalence is estimated toof the general population in the United StatesA shockingtoaccording to studiesof patients with AF are unaware of their diagnosissilent AFMost AF patients will suffer from congestive heart failure or and develop thromboembolic events leading to a dramatic reduction of patientsquality of lifeand a significant consumption of health care resource ...

Project SummaryDespite advances in the development of HIV drugs there remains a need for new therapiesToxicities associated with long term use of many of the approved HIV drugs coupled with the development of resistance drives the need for new and novel antiviralsMaturation inhibitorsMIsrepresent one such class of HIV therapiesHIV maturation inhibitors block virus replication by disrupting the con ...

SummaryThe goal of this proposal is to develop and validate a cost effectiveeasy to use and fully integrated navigation system for needle placement during CT image guided cancer diagnosis and treatmentBackgroundAccurate diagnostic and therapeutic interventions depend on accurate needle insertionwhich is subject to the physicianandapos s visuospatial ability and experienceand could be difficult in ...

Despite the myriad major advances in cardiologythe prognosis for patients with severemedically refractive heart failureHFis exceedingly poorApproximatelydeaths occur annually in patients with HF who have endured an impaired quality of life often accompanied by significant economic and personal losses due to recurrent hospitalizationsAt leastof these deaths were directly related to severe heart fai ...