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CHEMICALLY MODIFIED CELL-PENETRATING PEPTIDES FOR IMPROVED DELIVERY OF GENE MODULATING COMPOUNDS

机译:化学修饰的细胞穿透肽,可改善基因调节化合物的传递

摘要

PROBLEM TO BE SOLVED: To provide a system for intracellular cargo delivery comprising a new series of molecules that overcome the described drawbacks for non-covalent gene-delivery, ie low and heterogeneous delivery as well as toxicity, without requiring biocleavable linkers to cleave chloroquine analogues, and comprising irreversibly chloroquine-coupled compounds.SOLUTION: The present invention relates to a system for intracellular cargo delivery comprising at least one component A comprising at least one aliphatic linear or branched moiety with at least 4 carbon atoms, wherein the component A is attached to a cell penetrating peptide B which is selected from LLOOLAAAALOOLL [SEQ ID No 6], AGYLLGKLLOOLAAAALOOLL [SEQ ID No 2], INLKALAALAKKIL[SEQ ID No 28 ], and AGYLLGKINLKALAALAKKIL [SEQ ID No 1], and deletions, additions insertions and substitutions of amino acids.
机译:解决的问题:提供一种用于细胞内货物递送的系统,该系统包括一系列新的分子,这些分子克服了非共价基因递送的所述缺点,即低和异质递送以及毒性,而无需生物可裂解的接头裂解氯喹类似物。解决方案:本发明涉及一种用于细胞内货物递送的系统,该系统包括至少一种组分A,该组分A包含至少一个具有至少4个碳原子的脂族直链或支链部分,其中组分A连接至细胞穿透肽B,其选自LLOOLAAAALOOLL [SEQ ID No 6],AGYLLGKLLOOLAAAALOOLL [SEQ ID No 2],INLKALAALAKKIL [SEQ ID No 28]和AGYLLGKINLKALAALAKKIL [SEQ ID No 1],以及缺失,插入和取代氨基酸。

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