Reply

Martin Lagging, Karolina Rembeck, Jesper Waldenström, Staffan Nilsson, Kristina Nyström, Anna Martner, Magnus Lindh, Gunnar Norkrans, Johan Westin, Court Pedersen, Martti Färkkilä, Nina Langeland, Mads Rauning Buhl, Kristine Mørch, Peer B. Christensen, Kristoffer Hellstrand – 4 March 2014

Treatment of phenylketonuria using minicircle‐based naked‐DNA gene transfer to murine liver

Hiu Man Viecelli, Richard P. Harbottle, Suet Ping Wong, Andrea Schlegel, Marinee K. Chuah, Thierry VandenDriessche, Cary O. Harding, Beat Thöny – 28 February 2014 – Host immune response to viral vectors, persistence of nonintegrating vectors, and sustained transgene expression are among the major challenges in gene therapy. To overcome these hurdles, we successfully used minicircle (MC) naked‐DNA vectors devoid of any viral or bacterial sequences for the long‐term treatment of murine phenylketonuria, a model for a genetic liver defect.

Subscribe to