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Page 1
Widespread and sustained target engagement in Huntington's disease minipigs upon intrastriatal microRNA-based gene therapy.
Vallès A, Evers MM, Stam A, Sogorb-Gonzalez M, Brouwers C, Vendrell-Tornero C, Acar-Broekmans S, Paerels L, Klima J, Bohuslavova B, Pintauro R, Fodale V, Bresciani A, Liscak R, Urgosik D, Starek Z, Crha M, Blits B, Petry H, Ellederova Z, Motlik J, van Deventer S, Konstantinova P. Vallès A, et al. Among authors: petry h. Sci Transl Med. 2021 Apr 7;13(588):eabb8920. doi: 10.1126/scitranslmed.abb8920. Sci Transl Med. 2021. PMID: 33827977
A largely random AAV integration profile after LPLD gene therapy.
Kaeppel C, Beattie SG, Fronza R, van Logtenstein R, Salmon F, Schmidt S, Wolf S, Nowrouzi A, Glimm H, von Kalle C, Petry H, Gaudet D, Schmidt M. Kaeppel C, et al. Among authors: petry h. Nat Med. 2013 Jul;19(7):889-91. doi: 10.1038/nm.3230. Epub 2013 Jun 16. Nat Med. 2013. PMID: 23770691
Pathogenic classification of LPL gene variants reported to be associated with LPL deficiency.
Rodrigues R, Artieda M, Tejedor D, Martínez A, Konstantinova P, Petry H, Meyer C, Corzo D, Sundgreen C, Klor HU, Gouni-Berthold I, Westphal S, Steinhagen-Thiessen E, Julius U, Winkler K, Stroes E, Vogt A, Hardt P, Prophet H, Otte B, Nordestgaard BG, Deeb SS, Brunzell JD. Rodrigues R, et al. Among authors: petry h. J Clin Lipidol. 2016 Mar-Apr;10(2):394-409. doi: 10.1016/j.jacl.2015.12.015. Epub 2015 Dec 24. J Clin Lipidol. 2016. PMID: 27055971
46 results