Hepatocyte-directed gene transfer in vivo leads to transient improvement of hypercholesterolemia in low density lipoprotein receptor-deficient rabbits

J. M. Wilson, M. Grossman, C. H. Wu, N. R. Chowdhury, G. Y. Wu, J. R. Chowdhury

Research output: Contribution to journalArticle

202 Scopus citations


Familial hypercholesterolemia is an inherited disease in humans, caused by a deficiency of low density lipoprotein (LDL) receptors, that we have used as a model for developing liver-directed gene therapies. Our strategy is to reconstitute hepatic LDL receptor expression in vivo by administering a DNA-protein complex that is capable of targeting the delivery of functional LDL receptor genes to hepatocytes. Infusion of this DNA-protein complex into the peripheral circulation of a rabbit animal model for familial hypercholesterolemia resulted in hepatocyte-specific gene transfer and a temporary amelioration of hypercholesterolemia. This noninvasive approach to gene therapy should have applications in the treatment of a wide spectrum of human diseases.

Original languageEnglish (US)
Pages (from-to)963-967
Number of pages5
JournalJournal of Biological Chemistry
Issue number2
StatePublished - Jan 1 1992


ASJC Scopus subject areas

  • Biochemistry
  • Molecular Biology
  • Cell Biology

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