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Kassim SH, Wilson JM, Rader DJ. Gene therapy for dyslipidemia: a review of gene replacement and gene inhibition strategies. Clin Lipidol. 2010;5(6):793-809doi: 10.2217/clp.10.73.
Kassim, S. H., Wilson, J. M., & Rader, D. J. (2010). Gene therapy for dyslipidemia: a review of gene replacement and gene inhibition strategies. Clinical lipidology, 5(6), 793-809. https://doi.org/10.2217/clp.10.73
Kassim, Sadik H, et al. "Gene therapy for dyslipidemia: a review of gene replacement and gene inhibition strategies." Clinical lipidology vol. 5,6 (2010): 793-809. doi: https://doi.org/10.2217/clp.10.73
Kassim SH, Wilson JM, Rader DJ. Gene therapy for dyslipidemia: a review of gene replacement and gene inhibition strategies. Clin Lipidol. 2010 Jun;5(6):793-809. doi: 10.2217/clp.10.73. PMID: 22505953; PMCID: PMC3324780.
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Clin Lipidol. 2010 Jun;5(6):793-809. doi: 10.2217/clp.10.73.

Gene therapy for dyslipidemia: a review of gene replacement and gene inhibition strategies.

Clinical lipidology

Sadik H Kassim, James M Wilson, Daniel J Rader

Affiliations

  1. University of Pennsylvania School of Medicine, Gene Therapy Program, Department of Pathology & Laboratory Medicine, 125 South 31st Street (Suite 2000), PA 19104, USA.

PMID: 22505953 PMCID: PMC3324780 DOI: 10.2217/clp.10.73

Abstract

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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