mRNA therapy corrects defective glutathione metabolism and restores ureagenesis in preclinical argininosuccinic aciduria
mRNA疗法可纠正临床前精氨酸琥珀酸尿症中谷胱甘肽代谢缺陷并恢复尿素生成。
期刊:Science Translational Medicine
影响因子:14.6
doi:10.1126/scitranslmed.adh1334
Gurung, Sonam; Timmermand, Oskar Vilhelmsson; Perocheau, Dany; Gil-Martinez, Ana Luisa; Minnion, Magdalena; Touramanidou, Loukia; Fang, Sherry; Messina, Martina; Khalil, Youssef; Spiewak, Justyna; Barber, Abigail R; Edwards, Richard S; Pinto, Patricia Lipari; Finn, Patrick F; Cavedon, Alex; Siddiqui, Summar; Rice, Lisa; Martini, Paolo G V; Ridout, Deborah; Heywood, Wendy; Hargreaves, Ian; Heales, Simon; Mills, Philippa B; Waddington, Simon N; Gissen, Paul; Eaton, Simon; Ryten, Mina; Feelisch, Martin; Frassetto, Andrea; Witney, Timothy H; Baruteau, Julien