@article{ETD,
      school = {Ph.D.},
      author = {Richards, Daelyn Y.},
      url = {http://digitalcollections.ohsu.edu/record/8749},
      title = {Gene therapy advancements in murine phenylketonuria (PKU)},
      publisher = {Oregon Health and Science University},
      abstract = {The main focus of this dissertation work was to advance  gene therapy research for phenylketonuria (PKU). PKU is an  inborn error of metabolism characterized by phenylalanine  hydroxylase (PAH) deficiency that results in severe  neurological damage if left untreated. PAH functions in the  liver to metabolize the essential amino acid phenylalanine  (Phe), which is taken up in excess through dietary means.  The standard of care for PKU is a highly restricted diet  that is void of Phe that one must adhere to for life,  however the diet is challenging for all and unrealistic for  most. The PKU community has voiced their desire for the  development of a gene therapy that would, in essence, be a  one-time curative treatment for their disease, allowing for  a completely liberated diet without the devastating  neurological consequences.},
      number = {ETD},
      doi = {https://doi.org/10.6083/7p88ch091},
      recid = {8749},
      address = {2020},
}