GeneticsNature CommunicationsPeer-reviewed
Gene therapy in argininosuccinic aciduria
Julien Baruteau, Dany P.B. Perocheau, Joanna Hanley…·Simon N. Waddington
This paper demonstrates that gene therapy using AAV8 vector corrects urea cycle and citrulline-NO cycle defects in ASL-deficient mice, reducing neuronal disease and improving behavior.
47 citations