Safety and efficacy of an engineered hepatotropic AAV gene therapy for ornithine transcarbamylase deficiency in cynomolgus monkeys
Titel:
Safety and efficacy of an engineered hepatotropic AAV gene therapy for ornithine transcarbamylase deficiency in cynomolgus monkeys
Auteur:
Baruteau, Julien Cunningham, Sharon C. Yilmaz, Berna Seker Perocheau, Dany P. Eaglestone, Simon Burke, Derek Thrasher, Adrian J. Waddington, Simon N. Lisowski, Leszek Alexander, Ian E. Gissen, Paul