Novel AAV-mediated genome editing therapy improves health and survival in a mouse model of methylmalonic acidemia
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Shengwen Zhang | B. Chau | Matthias Hebben | Q. Xiong | Xiaohan Zhang | Susana Gordo | Chih-Wei Ko | Kyle Chiang | A. Bastille | Nikhil Ramesh | Jenisha Vora | Elizabeth McCarthy | Dylan Frank | Carmen Wu | Noel Walsh | Shreya Amarwani | Jing Liao | Lauren M. Drouin