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SYSTEMS AND METHODS OF DELIVERY OF BIOACTIVE AGENTS USING BACTERIAL TOXIN-DERIVED TRANSPORT SEQUENCES

申请号 US15837256 申请日 2017-12-11 公开(公告)号 US20180353610A1 公开(公告)日 2018-12-13
申请人 Applied Molecular Transport, LLC; 发明人 Randall Mrsny; Tahir Mahmood;
摘要 The field of the present invention relates, in part, to a strategy for novel pharmaceutical applications. More specifically, the present invention relates to a non-toxic mutant form of the Vibrio cholera Cholix gene (ntCholix), a variant of Cholix truncated at amino acid A386 (Cholix386) and the use of other various Cholix-derived polypeptide sequences to enhance intestinal delivery of biologically-active therapeutics. Importantly, the systems and methods described herein provide for the following: the ability to deliver macromolecule doses without injections; the ability to deliver cargo, such as (but not limited to) siRNA or antisense molecules into intracellular compartments where their activity is required; and the delivery of nanoparticles and dendrimer-based carriers across biological membranes, which otherwise would have been impeded due to the barrier properties of most such membranes.
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