Gene therapy in argininosuccinic aciduria
Julien Baruteau, Dany P.B. Perocheau, Joanna Hanley, Maëlle Lorvellec, Eridan Rocha‐Ferreira, Rajvinder Karda, Joanne Ng, Natalie Suff, Juan F. Antinao Diaz, Ahad A. Rahim, Michael P. Hughes, Blerida Banushi, Helen Prunty, Mariya Hristova, Deborah A. Ridout, , 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.
Nature Communications · Aug 23, 2018Peer-reviewedGenetics