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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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| Publicat a: | Gene Ther |
|---|---|
| Autors principals: | , , , , |
| Format: | Artigo |
| Idioma: | Inglês |
| Publicat: |
2014
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| Matèries: | |
| Accés en línia: | 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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