Gene therapy offers the possibility to treat pancreatic disease in Cystic Fibrosis (CF), caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene; however gene transfer to the pancreas is untested in humans. The pancreatic disease phenotype is very similar between humans and pigs with CF, thus CF pigs create an excellent opportunity to study gene transfer to the pancreas. There are no studies showing efficient transduction of pig pancreas with gene transfer vectors. Our objective is to develop a safe and efficient method to transduce wild-type (WT) porcine pancreatic ducts that express CFTR. We catheterized the umbilical artery of WT newborn pigs and delivered an adeno-associated virus serotype 9 vector expressing green fluorescent protein (AAV9CMV.sceGFP) or vehicle to the celiac artery, the vessel that supplies major branches to the pancreas. This technique resulted in stable and dose-dependent transduction of pancreatic duct epithelial cells that expressed CFTR. Intravenous injection of AAV9CMV.sceGFP did not transduce the pancreas. Our technique offers an opportunity to deliver the CFTR gene to the pancreas of CF pigs. The celiac artery can be accessed via umbilical artery in newborns and via femoral artery at older ages; delivery approaches which can be translated to humans.
challenges to be found for Pre-TED and Post-TED Forms were included in the new work flow. A list of diseasespecific staging was developed to guide disease status at annual evaluations. A visual approach was created in the spreadsheet to track forms completion with all patients due dates as follows: green-form may be completed, red-time to complete form has not yet been reached, blueform is ready to be reported, yellow-form must be reviewed, purple-patient underwent another HSCT and black-death. Conclusion: In October 2012 our goal was achieved and we were able to update and report all 193 patients. Team work and new efficient tools allowed control of due dates and optimization of time spent with data capturing, CRA/physician meetings and forms review. All items from all patients will now be timely reported.
Conclusions: 1) The process of donor clearance has become more complex and resource demanding. 2) Care should be taken in selecting donors for screening to avoid unnecessary added costs to the transplant.
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