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1.发明公开DELIVERY OF STRUCTURALLY DIVERSE POLYPEPTIDE CARGO INTO MAMMALIAN CELLS BY A BACTERIAL TOXIN 审中-公开细菌毒素将结构多样化的多肽货物运送到哺乳动物细胞中
公开(公告)号：EP3303403A1
公开(公告)日：2018-04-11
申请号：EP16802289.5
申请日：2016-05-31
IPC分类号： C07K19/00 , A61K35/74 , A61K38/16 , A61K38/45 , A61K38/48 , A61K47/48 , A61P25/00 , C07K14/34 , C12N15/09 , C12N15/62 , C12N9/10 , C12N9/48 , C12N9/52
CPC分类号： C12N15/62 , A61K35/74 , A61K38/00 , A61K38/45 , A61K38/48 , A61K47/42 , A61K47/6829 , C07K7/08 , C07K14/001 , C07K14/28 , C07K14/33 , C07K14/34 , C07K14/4703 , C07K14/475 , C07K16/1282 , C07K2319/10 , C07K2319/21 , C07K2319/22 , C07K2319/35 , C07K2319/41 , C07K2319/50 , C07K2319/55 , C07K2319/60 , C07K2319/75 , C12N9/1051 , C12N9/1077 , C12N9/22 , C12N9/2417 , C12N9/52 , C12N11/06 , C12N2310/20
摘要： There is a need for delivery platforms with robust capacity that offer the possibility to deliver diverse protein-based therapeutics into specific cells. Described herein is a platform for delivering cargo polypeptides into cells, which is based on a recombinant molecule comprising: a cargo polypeptide, a diphtheria toxin enzymatic fragment (DTA), and a diphtheria toxin translocation fragment (DTB). The platform has been employed to deliver diverse cargo into cells, including those having low or high molecular weights. A hyper-stable cargo polypeptide has been delivered, as well as proteins of therapeutic significance (e.g, MecP2, SMN, FMRP, PNP, alpha-amylase, and RRSP). The platform is also useful for delivering genome-modifying proteins, such as the CRISPR protein, Cas9. Associated nucleic acids, pharmaceutical compositions, methods, uses, and kits are also described, including those of therapeutic significance aimed at treating diseases or disorders caused by enzyme or protein deficiency.
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