Article
Therapeutic expression of hairpins targeting apolipoprotein B100 induces phenotypic and transcriptome changes in murine liver.
Gene therapy - 1 Jan 2014
Maczuga P, Verheij J, van der Loos C, van Logtenstein R, Hooijer G, Martier R, Borel F, Lubelski J, Koornneef A, Blits B, van Deventer S, Petry H, Konstantinova P
Abstract excerpt
Constitutive expression of short hairpin RNAs (shRNAs) may cause cellular toxicity in vivo and using microRNA (miRNA) scaffolds can circumvent this problem. Previously, we have shown that embedding small interfering RNA sequences targeting apolipoprotein B100 (ApoB) in shRNA (shApoB) or miRNA (miApoB) scaffolds resulted in differential processing and long-term efficacy in vivo. Here we show that adeno-associated...
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