Compositions and methods for treating mitochondrial neurogastrointestinal encephalopathy
申请人:Entrada Therapeutics, Inc.
公开号:US11168310B2
公开(公告)日:2021-11-09
Disclosed herein are compositions and methods of treating disclosure provides for compounds for use in treating Mitochondrial Neurogastrointestinal Encephalopathy Syndrome (MNGIE). In some embodiments, the compounds have cell penetrating activity and thymidine phosphorylase activity. In certain embodiments, the compounds disclosed herein comprise: a) at least one cell-penetrating peptide (CPP) moiety; and b) a thymidine phosphorylase, or an active fragment or analog thereof (TP), wherein the CPP is coupled, directly or indirectly, to TP.
本文公开了用于治疗线粒体神经胃肠道脑病综合征(MNGIE)的化合物的组合物和方法。在某些实施方案中,化合物具有细胞渗透活性和胸苷磷酸化酶活性。在某些实施方案中,本文公开的化合物包括:a)至少一种细胞穿透肽(CPP)分子;和b)胸苷磷酸化酶或其活性片段或类似物(TP),其中CPP直接或间接与TP偶联。