Long-term efficacy and safety of mRNA therapy in two murine models of methylmalonic acidemia

出版年份 2019 全文链接

标题

作者

关键词

Enzyme replacement, Liver targeted therapy, Methylmalonyl-CoA mutase, Organic acidemia, Systemic mRNA therapy

出版物

Volume 45, Issue -, Pages 519-528

出版商

Elsevier BV

发表日期

2019-07-12

DOI

10.1016/j.ebiom.2019.07.003

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