Rescue of glutaric aciduria type I in mice by liver-directed therapies
通过肝脏靶向疗法挽救小鼠戊二酸尿症 I 型
期刊:Science Translational Medicine
影响因子:15.8
doi:10.1126/scitranslmed.adf4086
Mercedes Barzi ,Collin G Johnson ,Tong Chen ,Ramona M Rodriguiz ,Madeline Hemmingsen ,Trevor J Gonzalez ,Alan Rosales ,James Beasley ,Cheryl K Peck ,Yunhan Ma ,Ashlee R Stiles ,Timothy C Wood ,Raquel Maeso-Diaz ,Anna Mae Diehl ,Sarah P Young ,Jeffrey I Everitt ,William C Wetsel ,William R Lagor ,Beatrice Bissig-Choisat ,Aravind Asokan ,Areeg El-Gharbawy ,Karl-Dimiter Bissig