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公开(公告)号：US20250090684A1
公开(公告)日：2025-03-20
申请号：US18292058
申请日：2022-07-26
Applicant: ModernaTX, Inc.
Inventor： Husain Attarwala , Jingsong Cao , Patrick Finn , Simone Mori
Abstract: This disclosure relates to mRNA therapy for the treatment of glycogen storage disease type 1a (GSD1a). mRNAs for use in the invention, when administered in vivo, encode glucose-6-phosphatase (G6PC). mRNA therapies of the disclosure increase and/or restore deficient levels of G6PC expression and/or activity in subjects.
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