@article{ETD,
      recid = {720},
      author = {Paulk, Nicole},
      title = {Novel strategies to improve viral gene targeting and  therapeutic liver repopulation in vivo},
      publisher = {Oregon Health and Science University},
      school = {Ph.D.},
      address = {2012},
      number = {ETD},
      abstract = {Orthotopic liver transplantation effectively treats  pediatric metabolic diseases caused by hepatocyte-specific  enzyme deficiencies, but donor scarcity, high cost, and  lifelong immunosuppression limit its use. This dissertation  explores alternative strategies: gene therapy and  hepatocyte transplantation. First, recombinant  adeno-associated viral (rAAV) vectors achieved stable in  vivo gene targeting in neonatal and adult mice, with  enhanced efficiency via transient inhibition of  non-homologous end joining using vanillin and proteasome  inhibition with bortezomib. Second, selective hepatocyte  repopulation was demonstrated using CEHPOBA, enabling  transient positive selection in wild-type livers. These  findings highlight promising approaches for correcting  metabolic liver disorders without whole-organ  transplantation.},
      url = {http://digitalcollections.ohsu.edu/record/720},
      doi = {https://doi.org/10.6083/M4K935JK},
}