Argininosuccinic aciduria fosters neuronal nitrosative stress reversed by Asl gene transfer

1 TITLE Argininosuccinic aciduria fosters neuronal nitrosative stress reversed by Asl gene transfer RUNNING TITLE Gene therapy in argininosuccinic aciduria AUTHORS Julien Baruteau 1,2,3 , Dany P. Perocheau 1 , Joanna Hanley 3,4 , Eridan Rocha-Ferreira 8 , Rajvinder Karda 1 , Joanne Ng 1,5 , Nattalie Suff 1 , Ahad A. Rahim 6 , Michael P. Hughes 6 , Blerida Banushi 4 , Helen Prunty 7 , Mariya Hristova 8 , Deborah A. Ridout 9 , Alex Virasami 10 , Simon Heales 3,7 , Stewen J. Howe 1 , Suzy M. Buckley 1 , Philippa B. Mills 3 , Paul Gissen 2,3,4 , Simon N. Waddington 1,11, * . AFFILIATIONS 1. Gene Transfer Technology Group, Institute for Women’s Health, University College London, 86-96 Chenies Mews, London WC1E 6HX, United Kingom. 2. Metabolic Medicine Department, Great Ormond Street Hospital for Children NHS Foundation Trust, London WC1N 3JH, United Kingom. . CC-BY-NC 4.0 International license available under a not certified by peer review) is the author/funder, who has granted bioRxiv a license to display the preprint in perpetuity. It is made The copyright holder for this preprint (which was this version posted June 15, 2018. ; https://doi.org/10.1101/348292 doi: bioRxiv preprint

Argininosuccinic aciduria fosters neuronal nitrosative stress reversed by Asl gene transfer

Others