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.
| Original language | English (US) |
|---|---|
| Pages (from-to) | 963-967 |
| Number of pages | 5 |
| Journal | Journal of Biological Chemistry |
| Volume | 267 |
| Issue number | 2 |
| State | Published - Jan 15 1992 |
| Externally published | Yes |
Fingerprint
ASJC Scopus subject areas
- Biochemistry
Cite this
Hepatocyte-directed gene transfer in vivo leads to transient improvement of hypercholesterolemia in low density lipoprotein receptor-deficient rabbits. / Wilson, James M.; Grossman, Mariann; Wu, Catherine H.; Chowdhury, N. Roy; Wu, George Y.; Roy-Chowdhury, Jayanta.
In: Journal of Biological Chemistry, Vol. 267, No. 2, 15.01.1992, p. 963-967.Research output: Contribution to journal › Article
}
TY - JOUR
T1 - Hepatocyte-directed gene transfer in vivo leads to transient improvement of hypercholesterolemia in low density lipoprotein receptor-deficient rabbits
AU - Wilson, James M.
AU - Grossman, Mariann
AU - Wu, Catherine H.
AU - Chowdhury, N. Roy
AU - Wu, George Y.
AU - Roy-Chowdhury, Jayanta
PY - 1992/1/15
Y1 - 1992/1/15
N2 - 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.
AB - 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.
UR - http://www.scopus.com/inward/record.url?scp=0026502766&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=0026502766&partnerID=8YFLogxK
M3 - Article
C2 - 1370472
AN - SCOPUS:0026502766
VL - 267
SP - 963
EP - 967
JO - Journal of Biological Chemistry
JF - Journal of Biological Chemistry
SN - 0021-9258
IS - 2
ER -