The DOI matches its Crossref record.
Hepatocyte-directed gene transfer in vivo leads to transient improvement of hypercholesterolemia in low density lipoprotein receptor-deficient rabbits.
Open accessAbstract
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.
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Cited by
Ishibashi, S., Brown, M. S., Goldstein, J. L., Gerard, R. D., Hammer, R. E., & Herz, J. (1993) · Journal of Clinical Investigation · doi:10.1172/jci116663 · Cited by 1,354
Gene therapy: adenovirus vectors
Kozarsky, K. F., & Wilson, J. M. (1993) · Current Opinion in Genetics & Development · doi:10.1016/0959-437x(93)90126-a · Cited by 419
Self-citationBenns, J. M., Choi, J.-S., Mahato, R. I., Park, J.-S., & Kim, S. W. (2000) · Bioconjugate Chemistry · doi:10.1021/bc0000177 · Cited by 381
Meier, M., Bider, M. D., Malashkevich, V. N., Spiess, M., & Burkhard, P. (2000) · Journal of Molecular Biology · doi:10.1006/jmbi.2000.3853 · Cited by 182
Receptor-mediated gene transfer vectors: progress towards genetic pharmaceuticals
Molas, M., Gomez-Valades, A., Vidal-Alabro, A., Miguel-Turu, M., Bermudez, J., Bartrons, R., & Perales, J. (2003) · Current Gene Therapy · doi:10.2174/1566523034578195 · Cited by 33
Metadata
- Authors
- Wilson, J. M., Grossman, M., Wu, C. H., Chowdhury, N. R., Wu, G. Y., & Chowdhury, J. R.
- Venue
- Journal of Biological Chemistry
- Publisher
- Elsevier BV
- Published
- 1992-01
- Volume
- 267
- Issue
- 2
- Pages
- 963-967
- ISSN
- 0021-9258 (ISSN-L)
- OpenAlex
- W1545653070
- Cited by
- 240
- References
- 0
- FWCI
- 12.22
- Type
- journal article
- Language
- en
- Open access
- hybrid
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- CoChat. (2026). Citation check: Hepatocyte-directed gene transfer in vivo leads to transient improvement of hypercholesterolemia in low density lipoprotein receptor-deficient rabbits. https://app.cochat.ai/papers/10.1016/s0021-9258(18)48379-2
- The paper
- Wilson, J. M., Grossman, M., Wu, C. H., Chowdhury, N. R., Wu, G. Y., & Chowdhury, J. R. (1992). Hepatocyte-directed gene transfer in vivo leads to transient improvement of hypercholesterolemia in low density lipoprotein receptor-deficient rabbits. Journal of Biological Chemistry. https://doi.org/10.1016/s0021-9258(18)48379-2
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Bibliographic data: Semantic Scholar, OpenAlex, Crossref.
Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).
Semantic Scholar’s data is described in: Kinney, R., et al. (2023). The Semantic Scholar Open Data Platform. arXiv:2301.10140.
