Correction of the genetic defect in hepatocytes from the Watanabe heritable hyperlipidemic rabbit.

@article{Wilson1988CorrectionOT,
  title={Correction of the genetic defect in hepatocytes from the Watanabe heritable hyperlipidemic rabbit.},
  author={J. M. Wilson and David J. Johnston and Douglas M. Jefferson and Richard C. Mulligan},
  journal={Proceedings of the National Academy of Sciences of the United States of America},
  year={1988},
  volume={85 12},
  pages={
          4421-5
        }
}
Familial hypercholesterolemia is an inherited disease in humans that is caused by a defect in the receptor for low density lipoproteins (LDLR). The existence of an animal model for this disease, the Watanabe heritable hyperlipidemic (WHHL) rabbit, makes it an attractive candidate for developing new therapies that involve gene transfer into liver. As a first step toward the development of these therapies, we report the use of retrovirus-mediated gene transfer to correct the genetic defect in… CONTINUE READING
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