Modeling correction of severe urea cycle defects in the growing murine liver using a hybrid recombinant adeno‐associated virus/piggyBac transposase gene delivery system
Sharon C. Cunningham, Susan M. Siew, Claus V. Hallwirth, Christine Bolitho, Natsuki Sasaki, Gagan Garg, Iacovos P. Michael, Nicola A. Hetherington, Kevin Carpenter, Gustavo de Alencastro, Andras Nagy, Ian E. Alexander – 9 April 2015 – Liver‐targeted gene therapy based on recombinant adeno‐associated viral vectors (rAAV) shows promising therapeutic efficacy in animal models and adult‐focused clinical trials.