An AAV-SGCG Dose-Response Study in a Γ-Sarcoglycanopathy Mouse Model in the Context of Mechanical Stress
Molecular therapy Methods & clinical development（2019）
关键词
LGMD2C,γ-sarcoglycanopathy,limb girdle muscular dystrophy,gene therapy,translational medicine,AAV,miRNA biomarker,mechanical stress
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