Transplantation of genetically modified autologous hepatocytes into nonhuman primates: feasibility and short-term toxicity.

@article{Grossman1992TransplantationOG,
  title={Transplantation of genetically modified autologous hepatocytes into nonhuman primates: feasibility and short-term toxicity.},
  author={Mariann Grossman and Steven E. Raper and James Michael Wilson},
  journal={Human gene therapy},
  year={1992},
  volume={3 5},
  pages={501-10}
}
Ex vivo gene therapy directed to the liver is being developed for the treatment of inherited metabolic diseases. Transplantation of hepatocytes that have been transduced with a low-density lipoprotein (LDL) receptor gene is a potential form of therapy for familial hypercholesterolemia (FH). We have demonstrated efficacy of ex vivo gene therapy for familial hypercholesterolemia in a rabbit animal model of this disease. In preparation for human trials, we describe in this report experiments in… CONTINUE READING

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