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PROGRAMMABLE DESIGNER THERAPEUTIC FUSOGENIC SECRETED GECTOSOME VESICLES FOR MACROMOLECULE DELIVERY AND GENOME MODIFICATION

机译:用于大分子传递和基因组修饰的可编程设计的治疗性,遗传性分泌型核糖体囊泡

摘要

The invention includes systems, methods, and compositions for designing secreted fusogenic ectosome vesicles, or gectosomes, that selectively encapsulate specific target proteins, nucleic acids and/or other small molecules in a predetermined manner. These engineered gectosomes can be used to deliver desired cargos to receipt cells in vitro, ex vivo, or in vivo and may further reprogram target cellular phenotypes in a dose-dependent manner, as well as perform genome editing functions among others.
机译:本发明包括用于设计分泌的融合的囊泡或胶体的系统,方法和组合物,其以预定的方式选择性地包封特定的靶蛋白,核酸和/或其他小分子。这些工程化的小体可用于在体外,离体或体内将所需的货物递送至接收细胞,并且可以以剂量依赖性方式进一步重编程靶细胞表型,以及执行基因组编辑功能。

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