Publication Date:
1991-12-30
Description:
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.〈br /〉〈span class="detail_caption"〉Notes: 〈/span〉Chowdhury, J R -- Grossman, M -- Gupta, S -- Chowdhury, N R -- Baker, J R Jr -- Wilson, J M -- P01-DK-42718/DK/NIDDK NIH HHS/ -- R01-DK-34357/DK/NIDDK NIH HHS/ -- R01-DK42193-01/DK/NIDDK NIH HHS/ -- etc. -- New York, N.Y. -- Science. 1991 Dec 20;254(5039):1802-5.〈br /〉〈span class="detail_caption"〉Author address: 〈/span〉Department of Internal Medicine, Albert Einstein College of Medicine, Bronx, NY 10461.〈br /〉〈span class="detail_caption"〉Record origin:〈/span〉 〈a href="http://www.ncbi.nlm.nih.gov/pubmed/1722351" target="_blank"〉PubMed〈/a〉
Keywords:
Animals
;
Gene Expression
;
*Genetic Therapy
;
Hypercholesterolemia/*genetics/*therapy
;
Liver/physiology
;
Liver Transplantation/physiology
;
RNA/genetics/isolation & purification
;
Rabbits
;
Receptors, LDL/analysis/*genetics
;
Recombinant Proteins/analysis
;
Serum Albumin/analysis/genetics
;
*Transfection
;
beta-Galactosidase/analysis/genetics
Print ISSN:
0036-8075
Electronic ISSN:
1095-9203
Topics:
Biology
,
Chemistry and Pharmacology
,
Computer Science
,
Medicine
,
Natural Sciences in General
,
Physics
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