Gene therapy for rhesus monkeys heterozygous for LDL receptor deficiency by balloon-catheter hepatic delivery of helper-dependent adenoviral vector

Autosomal dominant familial hypercholesterolemia (FH) is a monogenic life-threatening disease. We tested the efficacy of low-density lipoprotein receptor (LDLR) gene therapy using helper-dependent adenoviral vector (HDAd) in a nonhuman primate model of FH, comparing intravenous injection versus intr...

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Pubblicato in:Gene Ther
Autori principali: Oka, Kazuhiro, Mullins, Charles E., Kushwaha, Rampratap S., Leen, Ann M, Chan, Lawrence
Natura: Artigo
Lingua:Inglês
Pubblicazione: 2014
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Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC4289097/
https://ncbi.nlm.nih.gov/pubmed/25231173
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gt.2014.85
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