Gene therapy in a humanized mouse model of familial hypercholesterolemia leads to marked regression of atherosclerosis.

<h4>Background</h4>Familial hypercholesterolemia (FH) is an autosomal codominant disorder caused by mutations in the low-density lipoprotein receptor (LDLR) gene. Homozygous FH patients (hoFH) have severe hypercholesterolemia leading to life threatening atherosclerosis in childhood and a...

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Autores principales: Sadik H Kassim, Hui Li, Luk H Vandenberghe, Christian Hinderer, Peter Bell, Dawn Marchadier, Aisha Wilson, Debra Cromley, Valeska Redon, Hongwei Yu, James M Wilson, Daniel J Rader
Formato: article
Lenguaje:EN
Publicado: Public Library of Science (PLoS) 2010
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Acceso en línea:https://doaj.org/article/e79587d53a7b4820b632c5dfd2a0f483
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