Exon Skipping of Hepatic APOB Pre-mRNA With Splice-switching Oligonucleotides Reduces LDL Cholesterol In Vivo

Familial hypercholesterolemia (FH) is a genetic disorder characterized by extremely high levels of plasma low-density lipoprotein (LDL), due to defective LDL receptor-apolipoprotein B (APOB) binding. Current therapies such as statins or LDL apheresis for homozygous FH are insufficiently efficacious...

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Autores principales: Disterer, Petra, Al-Shawi, Raya, Ellmerich, Stephan, Waddington, Simon N, Owen, James S, Simons, J Paul, Khoo, Bernard
Formato: Artigo
Lenguaje:Inglês
Publicado: Nature Publishing Group 2013
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Original Article
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC3589156/
https://ncbi.nlm.nih.gov/pubmed/23319054
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2012.264
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