A novel screening system improves genetic correction by internal exon replacement

by Koller, Ulrich, Wally, Verena, Mitchell, Lloyd G., Klausegger, Alfred, Murauer, Eva M., Mayr, Elisabeth, Gruber, Christina, Hainzl, Stefan, Hintner, Helmut, Bauer, Johann W.
Published 2011

Development of Polypeptide-based Nanoparticles for Non-viral Delivery of CD22 RNA Trans-splicing Molecule as a New Precision Medicine Candidate Against B-lineage ALL

by Uckun, Fatih M., Mitchell, Lloyd G., Qazi, Sanjive, Liu, Yang, Zheng, Nan, Myers, Dorothea E., Song, Ziyuan, Ma, Hong, Cheng, Jianjun
Published 2015