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  • 标题:Gene therapy for lipid disorders
  • 本地全文:下载
  • 作者:Masa-aki Kawashiri ; Daniel J Rader
  • 期刊名称:Trials
  • 印刷版ISSN:1745-6215
  • 电子版ISSN:1745-6215
  • 出版年度:2000
  • 卷号:1
  • 期号:2
  • 页码:120
  • DOI:10.1186/cvm-1-2-120
  • 语种:English
  • 出版社:BioMed Central
  • 摘要:

    Lipid disorders are associated with atherosclerotic vascular disease, and therapy is associated with a substantial reduction in cardiovascular events. Current approaches to the treatment of lipid disorders are ineffective in a substantial number of patients. New therapies for refractory hypercholesterolemia, severe hypertriglyceridemia, and low levels of high-density lipoprotein cholesterol are needed: somatic gene therapy is one viable approach. The molecular etiology and pathophysiology of most of the candidate diseases are well understood. Animal models exist for the diseases and in many cases preclinical proof-of-principle studies have already been performed. There has been progress in the development of vectors that provide long-term gene expression. New clinical gene therapy trials for lipid disorders are likely to be initiated within the next few years.

  • 关键词:atherosclerosis; cholesterol; gene therapy; lipoproteins; vectors
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