期刊
CLINICAL LIPIDOLOGY
卷 5, 期 6, 页码 793-809出版社
FUTURE MEDICINE LTD
DOI: 10.2217/CLP.10.73
关键词
dyslipidemia; gene therapy; lipoprotein
资金
- National Heart Lung and Blood Institute [P01-HL059407]
- National Institute of Diabetes and Digestive and Kidney Diseases [P30-DK047757]
Despite numerous technological and pharmacological advances and more detailed knowledge of molecular etiologies, cardiovascular diseases remain the leading cause of morbidity and mortality worldwide claiming over 17 million lives a year. Abnormalities in the synthesis, processing and catabolism of lipoprotein particles can result in severe hypercholesterolemia, hypertriglyceridemia or low HDL-C. Although a plethora of antidyslipidemic pharmacological agents are available, these drugs are relatively ineffective in many patients with Mendelian lipid disorders, indicating the need for new and more effective interventions. In vivo somatic gene therapy is one such intervention. This article summarizes current strategies being pursued for the development of clinical gene therapy for dyslipidemias that cannot effectively be treated with existing drugs.
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