新化合物能修复RNA异常有助开发罕见病新疗法

金融界2025-04-15

日本京都大学等机构研究人员在新一期美国《科学进展》杂志上发表论文说，他们研发出一种低分子化合物，口服后能修复RNA(核糖核酸)错误剪接，从而为治疗法布雷病等基因突变引发的罕见遗传病提供新思路。研究人员说，这一成果不只是局限于法布雷病，也有可能应用于RNA错误剪接所导致的其他遗传病。这种化合物可以口服并有效地递送到心脏组织，有望在症状出现前预防性治疗相关疾病。责任编辑...

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新化合物能修复RNA异常 有助开发罕见病新疗法

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新化合物能修复RNA异常有助开发罕见病新疗法