The primary goal of the human applications core is to maximize the safety, validity and efficiency of introducing gene therapy into human trials involving patients with cystic fibrosis and other genetic disorders. The objectives of the core are to 1) collaborate with investigations within the gene therapy core center in design and implementation of preclinical studies to assure that necessary safety and efficacy data have been collected prior to initiation of human trials, 2) provide enrollment, study implementation, data collection, and analyses for gene therapy trials. In the previous grant cycle, the core completed 2 human trials, one involving repeat adenoviral delivery of the CFTR cDNA to nasal epithelia in patients with CF and a second involving retroviral mediated ex vivo transduction of glucocerebrosidase cDNA into peripheral blood repopulating cells (PRBC's) with subsequent readministration to patients with Gaucher's Disease. In the renewal period (1999-2004), the human core will initiate three proposed human trials: 1) AAV mediated delivery of a marker gene, human placental alkaline phosphoration to respiratory epithelium of patients with CF, 2) Ex vivo transduction of retroviral mediated human CD18 cDNA to PBRC's of patients with LAD, and Ex vivo retroviral mediated transfer of human erythropoietin (EPO) cDNA into vascular smooth muscle cells from patients with end stage renal disease (ESRD) seemed into a prosthetic access graft for treatment of anemia in individuals requiring hemodialysis. In summary, the benefit of the core is to consolidate resources and assure that human trials are designed and conducted properly to answer proposed scientific questions.
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