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Systemic Messenger RNA (mRNA) Therapy Delivered via Lipid Nanoparticle as a Treatment for MUT Class Methylmalonic Acidemia (MMA)

Jessica L. Schneller, Jeff An,Andrea Frassetto,Shi Liang,Xuling Zhu,Ji-Sun Park,Sue-Jean Hong,Jenny Zhuo,Raj Rajendran,Vladimir Presnyak,Staci Sabnis,Christine Lukacs,Lin Guey,Paolo Martini,Randy J. Chandler,Charles P. Venditti

MOLECULAR THERAPY（2017）

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