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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Detalles Bibliográficos
Autores principales: Oka, Kazuhiro, Mullins, Charles E., Kushwaha, Rampratap S., Leen, Ann M, Chan, Lawrence
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2014
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4289097/
https://www.ncbi.nlm.nih.gov/pubmed/25231173
http://dx.doi.org/10.1038/gt.2014.85

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