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| HEPATOCYTE-DIRECTED GENE-TRANSFER INVIVO LEADS TO TRANSIENT IMPROVEMENT OF HYPERCHOLESTEROLEMIA IN LOW-DENSITY-LIPOPROTEIN RECEPTOR-DEFICIENT RABBITS |
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| Author(s): WILSON JM, GROSSMAN M, WU CH, CHOWDHURY NR, WU GY, CHOWDHURY JR |
| Source: JOURNAL OF BIOLOGICAL CHEMISTRY Volume: 267 Issue: 2 Pages: 963-967 Published: JAN 15 1992 |
| Times Cited: 190 References: 21 |
| Abstract: 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. |
| Document Type: Article |
| Language: English |
| Reprint Address: WILSON, JM (reprint author), UNIV MICHIGAN, HOWARD HUGHES MED INST, DEPT INTERNAL MED, ANN ARBOR, MI 48109 USA |
Addresses:
1. UNIV MICHIGAN, HOWARD HUGHES MED INST, DEPT BIOL CHEM, ANN ARBOR, MI 48109 USA
2. UNIV CONNECTICUT, SCH MED, DEPT INTERNAL MED, FARMINGTON, CT 06030 USA
3. YESHIVA UNIV ALBERT EINSTEIN COLL MED, LIVER RES CTR, DEPT INTERNAL MED, BRONX, NY 10461 USA |
| Publisher: AMER SOC BIOCHEMISTRY MOLECULAR BIOLOGY INC, 9650 ROCKVILLE PIKE, BETHESDA, MD 20814 |
| Subject Category: Biochemistry & Molecular Biology |
| IDS Number: GY960 |
| ISSN: 0021-9258 |
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