Long-term improvement of hypercholesterolemia after ex vivo gene therapy in LDLR-deficient rabbits

Jayanta Roy-Chowdhury, Mariann Grossman, Sanjeev Gupta, N. Roy Chowdhury, James R. Baker, James M. Wilson

Research output: Contribution to journalArticle

305 Citations (Scopus)

Abstract

Familial hypercholesterolemia (FH) is an inherited disorder in humans that is caused by a deficiency of low density lipoprotein receptors (LDLRs). An animal model for FH, the Watanabe Heritable Hyperlipidemic rabbit, was used to develop an approach for liver-directed gene therapy based on transplantation of autologous hepatocytes that were genetically corrected ex vivo with recombinant retroviruses. Animals transplanted with LDLR-transduced autologous hepatocytes demonstrated a 30 to 50 percent decrease in total serum cholesterol that persisted for the duration of the experiment (122 days). Recombinant-derived LDLR RNA was harvested from tissues with no diminution for up to 6.5 months after transplantation.

Original languageEnglish (US)
Pages (from-to)1802-1805
Number of pages4
JournalScience
Volume254
Issue number5039
StatePublished - Dec 20 1991

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LDL Receptors
Hypercholesterolemia
Genetic Therapy
Hyperlipoproteinemia Type II
Rabbits
Hepatocytes
Autologous Transplantation
Retroviridae
Animal Models
Transplantation
Cholesterol
RNA
Liver
Serum

ASJC Scopus subject areas

  • General

Cite this

Roy-Chowdhury, J., Grossman, M., Gupta, S., Chowdhury, N. R., Baker, J. R., & Wilson, J. M. (1991). Long-term improvement of hypercholesterolemia after ex vivo gene therapy in LDLR-deficient rabbits. Science, 254(5039), 1802-1805.

Long-term improvement of hypercholesterolemia after ex vivo gene therapy in LDLR-deficient rabbits. / Roy-Chowdhury, Jayanta; Grossman, Mariann; Gupta, Sanjeev; Chowdhury, N. Roy; Baker, James R.; Wilson, James M.

In: Science, Vol. 254, No. 5039, 20.12.1991, p. 1802-1805.

Research output: Contribution to journalArticle

Roy-Chowdhury, J, Grossman, M, Gupta, S, Chowdhury, NR, Baker, JR & Wilson, JM 1991, 'Long-term improvement of hypercholesterolemia after ex vivo gene therapy in LDLR-deficient rabbits', Science, vol. 254, no. 5039, pp. 1802-1805.
Roy-Chowdhury, Jayanta ; Grossman, Mariann ; Gupta, Sanjeev ; Chowdhury, N. Roy ; Baker, James R. ; Wilson, James M. / Long-term improvement of hypercholesterolemia after ex vivo gene therapy in LDLR-deficient rabbits. In: Science. 1991 ; Vol. 254, No. 5039. pp. 1802-1805.
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