Correction of feline lipoprotein lipase deficiency with adeno-associated virus serotype 1-mediated gene transfer of the lipoprotein lipase S447X beneficial mutation.

@article{Ross2006CorrectionOF,
  title={Correction of feline lipoprotein lipase deficiency with adeno-associated virus serotype 1-mediated gene transfer of the lipoprotein lipase S447X beneficial mutation.},
  author={Colin J. D. Ross and Jaap Twisk and Andrew C. Bakker and Fudan Miao and Dennis Verbart and Jaap Rip and Tamara Godbey and Paul A Dijkhuizen and Wim T J M C Hermens and John J. P. Kastelein and Jan A. Kuivenhoven and Janneke J M Meulenberg and Michael R. Hayden},
  journal={Human gene therapy},
  year={2006},
  volume={17 5},
  pages={487-99}
}
Human lipoprotein lipase (hLPL) deficiency, for which there currently exists no adequate treatment, leads to excessive plasma triglycerides (TGs), recurrent abdominal pain, and life-threatening pancreatitis. We have shown that a single intramuscular administration of adeno-associated virus (AAV) serotype 1 vector, encoding the human LPL(S447X) variant, results in complete, long-term normalization of dyslipidemia in LPL(/) mice. As a prelude to gene therapy for human LPL deficiency, we tested… CONTINUE READING

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