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AAV-mediated in vivo knockdown of luciferase using combinatorial RNAi and U1i.
Koornneef A, van Logtenstein R, Timmermans E, Pisas L, Blits B, Abad X, Fortes P, Petry H, Konstantinova P, Ritsema T. Koornneef A, et al. Among authors: petry h. Gene Ther. 2011 Sep;18(9):929-35. doi: 10.1038/gt.2011.41. Epub 2011 Apr 7. Gene Ther. 2011. PMID: 21472008 Free PMC article.
Therapeutic expression of hairpins targeting apolipoprotein B100 induces phenotypic and transcriptome changes in murine liver.
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. Maczuga P, et al. Among authors: petry h. Gene Ther. 2014 Jan;21(1):60-70. doi: 10.1038/gt.2013.58. Epub 2013 Oct 24. Gene Ther. 2014. PMID: 24152580 Free PMC article.
AAV5-miHTT Gene Therapy Demonstrates Broad Distribution and Strong Human Mutant Huntingtin Lowering in a Huntington's Disease Minipig Model.
Evers MM, Miniarikova J, Juhas S, Vallès A, Bohuslavova B, Juhasova J, Skalnikova HK, Vodicka P, Valekova I, Brouwers C, Blits B, Lubelski J, Kovarova H, Ellederova Z, van Deventer SJ, Petry H, Motlik J, Konstantinova P. Evers MM, et al. Among authors: petry h. Mol Ther. 2018 Sep 5;26(9):2163-2177. doi: 10.1016/j.ymthe.2018.06.021. Epub 2018 Jun 25. Mol Ther. 2018. PMID: 30007561 Free PMC article.
Embedding siRNA sequences targeting apolipoprotein B100 in shRNA and miRNA scaffolds results in differential processing and in vivo efficacy.
Maczuga P, Lubelski J, van Logtenstein R, Borel F, Blits B, Fakkert E, Costessi A, Butler D, van Deventer S, Petry H, Koornneef A, Konstantinova P. Maczuga P, et al. Among authors: petry h. Mol Ther. 2013 Jan;21(1):217-27. doi: 10.1038/mt.2012.160. Epub 2012 Oct 23. Mol Ther. 2013. PMID: 23089734 Free PMC article.
AAV5-miHTT gene therapy demonstrates suppression of mutant huntingtin aggregation and neuronal dysfunction in a rat model of Huntington's disease.
Miniarikova J, Zimmer V, Martier R, Brouwers CC, Pythoud C, Richetin K, Rey M, Lubelski J, Evers MM, van Deventer SJ, Petry H, Déglon N, Konstantinova P. Miniarikova J, et al. Among authors: petry h. Gene Ther. 2017 Oct;24(10):630-639. doi: 10.1038/gt.2017.71. Epub 2017 Aug 3. Gene Ther. 2017. PMID: 28771234 Free PMC article.
229 results