科学家开发“StitchR”基因疗法,以有效治疗动物模型中的肌肉萎缩症。 Scientists develop "StitchR" gene therapy to effectively treat muscular dystrophy in animal models.

科学家开发了一种叫做“StitchR”的新型基因治疗方法,用于治疗肌肉萎缩症。 Scientists have developed a new gene therapy method called "StitchR" to treat muscular dystrophy. 该技术分两半提供大型治疗基因,然后将细胞内部结合以恢复缺失的蛋白质。 The technology delivers large therapeutic genes in two halves, which then join together inside cells to restore missing proteins. 它成功地恢复了肌肉萎缩动物模型中的正常蛋白质水平,克服了以前基因疗法中无法有效提供大型基因的局限性。 It successfully restored normal protein levels in animal models of muscular dystrophy, overcoming limitations of previous gene therapies that could not deliver large genes effectively.