Gene Therapy in a Humanized Mouse Model of Familial Hypercholesterolemia Leads to Marked Regression of Atherosclerosis

BACKGROUND: 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 adolescence. Mice wi...

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Autori principali: Kassim, Sadik H., Li, Hui, Vandenberghe, Luk H., Hinderer, Christian, Bell, Peter, Marchadier, Dawn, Wilson, Aisha, Cromley, Debra, Redon, Valeska, Yu, Hongwei, Wilson, James M., Rader, Daniel J.
Natura: Artigo
Lingua:Inglês
Pubblicazione: Public Library of Science 2010
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Research Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC2957433/
https://ncbi.nlm.nih.gov/pubmed/20976059
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0013424
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