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1.发明申请公开(公告)号：US20220370354A1
公开(公告)日：2022-11-24
申请号：US17608691
申请日：2020-05-08
Applicant: ModernaTX, Inc.
Inventor： Tal Zaks , Kelly Lindert , Lin Tung Guey
IPC: A61K9/127 , A61K31/7115 , A61K9/00 , A61K9/51 , A61P3/00
Abstract: This disclosure relates to mRNA therapy for the treatment of methylmalonic acidemia (MMA). mRNAs for use in the invention, when administered in vivo, encode methylmalonyl-CoA mutase (MUT). mRNA therapies of the disclosure increase and/or restore deficient levels of MUT expression and/or activity in subjects.
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