Therapeutic expression of hairpins targeting apolipoprotein B100 induces phenotypic and transcriptome changes in murine liver

由 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
出版 2014

AAV5-miHTT gene therapy demonstrates suppression of mutant huntingtin aggregation and neuronal dysfunction in a rat model of Huntington’s disease

由 Miniarikova, J, Zimmer, V, Martier, R, Brouwers, C C, Pythoud, C, Richetin, K, Rey, M, Lubelski, J, Evers, M M, van Deventer, S J, Petry, H, Déglon, N, Konstantinova, P
發表在 Gene Ther (2017)