申请人:The Regents of the University of California
公开号:US20180086782A1
公开(公告)日:2018-03-29
This disclosure relates to the incorporation of amphiphilic guanidinylated aminoglycosides (e.g., neomycin) into liposomal assemblies, which contain entrapped therapeutics. The lysosome is responsible for enzymatically breaking down and recycling large biomolecules and aged organelles. While malfunctioned lysosomal enzymes have been established in Lysosomal Storage Disorders (LSDs), recent reports have suggested that defects in lysosomal enzymes (e.g., glucocerebrosidase) are also linked to other chronic ailments, including neurological disorders such as Parkinson's Disease and related disorders.
本公开涉及将两性亲和性胍基化氨基糖苷(例如新霉素)纳入含有封闭治疗剂的脂质体聚集体中。溶酶体负责酶解和回收大分子生物分子和老化的细胞器。虽然已经确定溶酶体酶在溶酶体贮积病(LSDs)中存在故障,但最近的报告表明,溶酶体酶(例如葡萄糖鞘脂酶)的缺陷也与其他慢性疾病有关,包括神经疾病如帕金森病及相关疾病。