作者
Xianjun Gao, Ning Ran, Xue Dong, Bingfeng Zuo, Rong Yang, Qibing Zhou, Hong M Moulton, Yiqi Seow, HaiFang Yin
发表日期
2018/6/6
期刊
Science translational medicine
卷号
10
期号
444
页码范围
eaat0195
出版商
American Association for the Advancement of Science
简介
Exosomes are circulating nanovesicular carriers of macromolecules, increasingly used for diagnostics and therapeutics. The ability to load and target patient-derived exosomes without altering exosomal surfaces is key to unlocking their therapeutic potential. We demonstrate that a peptide (CP05) identified by phage display enables targeting, cargo loading, and capture of exosomes from diverse origins, including patient-derived exosomes, through binding to CD63—an exosomal surface protein. Systemic administration of exosomes loaded with CP05-modified, dystrophin splice–correcting phosphorodiamidate morpholino oligomer (EXOPMO) increased dystrophin protein 18-fold in quadriceps of dystrophin-deficient mdx mice compared to CP05-PMO. Loading CP05-muscle–targeting peptide on EXOPMO further increased dystrophin expression in muscle with functional improvement without any detectable toxicity …
引用总数
20182019202020212022202320245213763746646
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