Codon-optimized human Smad7 gene therapy enhances skeletal muscle mass and function in a murine model of Duchenne muscular dystrophy
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来源：
Nature
关键字：
computational pathology
发布时间：
2025-11-26 03:47
摘要：
AVGN7.2, a codon-optimized human Smad7 gene therapy, has shown significant potential in enhancing skeletal muscle mass and function in murine models of Duchenne muscular dystrophy (DMD). The therapy demonstrated improvements in muscle hypertrophy and function without exacerbating disease symptoms. The study highlights the importance of codon optimization in gene therapy, suggesting that AVGN7.2 could be a promising treatment for muscle wasting diseases, paving the way for future clinical applications.
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关键证据
AVGN7.2 significantly increased muscle mass and function in murine models of Duchenne muscular dystrophy.
Codon optimization improved the therapeutic efficacy of hSmad7.
The study suggests AVGN7.2 could be a viable treatment option for muscle wasting diseases.
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AI评分总结
AVGN7.2, a codon-optimized human Smad7 gene therapy, has shown significant potential in enhancing skeletal muscle mass and function in murine models of Duchenne muscular dystrophy (DMD). The therapy demonstrated improvements in muscle hypertrophy and function without exacerbating disease symptoms. The study highlights the importance of codon optimization in gene therapy, suggesting that AVGN7.2 could be a promising treatment for muscle wasting diseases, paving the way for future clinical applications.