申请人:Marker Gene Technologies, Inc.
公开号:US10543282B2
公开(公告)日:2020-01-28
The present invention relates to the preparation and use of therapeutic compounds for the treatment of diseases at specific subcellular target areas such as specific cellular organelles. In particular, the therapeutic compounds of the invention are specific for modifying enzyme activity within targeted organelles or structures of cells and tissues. Subcellular organelles and structures that may be specifically targeted by compounds of the present invention include lysosomes, autophagasomes, the endoplasmic reticulum, the Golgi complex, peroxisomes, the nucleus, membranes and the mitochondria.
本发明涉及用于治疗特定亚细胞靶区(如特定细胞器)疾病的治疗化合物的制备和使用。特别是,本发明的治疗性化合物对改变细胞和组织的目标细胞器或结构内的酶活性具有特异性。本发明化合物可特异性靶向的亚细胞器和结构包括溶酶体、自噬体、内质网、高尔基复合体、过氧物酶体、细胞核、细胞膜和线粒体。