Accelerated Endosomal Escape of Splice-Switching Oligonucleotides Enables Efficient Hepatic Splice Correction.

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作者： Silvia,Weiss ^[1] ; Simon,Decker ^[1] ; Christoph,Kugler ^[1] ; Laura Bocanegra,Gómez ^[1] ; Helene,Fasching ^[1] ; Denise,Benisch ^[1] ; Fatih,Alioglu ^[1] ; Levente,Ferencz ^[1] ; Theresa,Birkfeld ^[1] ; Filip,Ilievski ^[1] ; Volker,Baumann ^[1] ; Alina,Duran ^[1] ; Enes,Dusinovic ^[1] ; Nadine,Follrich ^[1] ; Sandra,Milenkovic ^[1] ; Dajana,Mihalicokova ^[1] ; Daniel,Paunov ^[1] ; Karla,Singeorzan ^[1] ; Nikolaus,Zehetmayer ^[1] ; Dejan,Zivanonvic ^[1] ; Ulrich,Lächelt ^[1] ; Auke,Boersma ^[2] ; Thomas,Rülicke ^[3] ; Haider,Sami ^[1] ; Manfred,Ogris ^[1]

作者单位： Faculty of Life Sciences, Department of Pharmaceutical Sciences, Laboratory of Macromolecular Cancer Therapeutics (MMCT), University of Vienna, Josef-Holaubek-Platz 2, 1090 Vienna, Austria. ^[1] Institute of In-Vivo and In-Vitro Models, Biomodels Austria, Department of Biomedical Sciences, University of Veterinary Medicine Vienna, Veterinärplatz 1, A-1210 Vienna, Austria. ^[2] Department of Biomedical Sciences and Pathobiology, University of Veterinary Medicine Vienna and Ludwig Boltzmann Institute for Hematology and Oncology, Veterinärplatz 1, A-1210 Vienna, Austria. ^[3]

关键词 biological barriers crosslinked polyethylenimine endosomal escape intravenous optical tomography phosphorothioate splice correction