CHUNG, Jong Hoon,정종훈,GARG, Pankaj,가르그반가지,PANDEY, Shambhavi,팬디샴하비,CHOUNG, Pill Hoon,정필훈,CHO, Chong Su,조종수,KIM, Bog Hee,김복희
申请号：
KRKR2015/011494
公开号：
WO2016/068617A1
申请日:
2015.10.29
申请国别(地区):
WO
年份:
2016
代理人:
摘要：
The present invention relates to polydixylitol polymer based gene transporter (PdXYP) and a method for producing the same. In addition, the present invention relates to a nucleic acid transport complex in which a therapeutic nucleic acid is bound to the gene transporter, and to a pharmaceutical composition for genetic therapy containing the corresponding complex as an active ingredient. Furthermore, the present invention relates to the gene transporter, a gene transport complex, and a gene therapy using the same. It was verified that the polydixylitol polymer based gene transporter (PdXYP) of the present invention has a remarkably higher nucleic acid transport rate than existing nucleic acid transporters, little cytotoxicity of a conjugate when being conjugated with DNA, very high in vivo transformation efficiency, and a remarkably high transformation efficiency in brain tissues, which have had difficulty in genetic therapy due to blood brain barriers. Hence, the gene transporter of the present invention can be widely used as an experimental gene transporter as well as in a field of genetic therapy for various diseases according to therapeutic nucleic acids binding to the gene transporter in various tissues in vivo.La présente invention concerne un transporteur de gènes à base de polymère polydixylitol (PdXYP) et un procédé de production de celui-ci. En outre, la présente invention concerne un complexe de transport dacide nucléique dans lequel un acide nucléique thérapeutique est lié au transporteur de gènes, et une composition pharmaceutique pour thérapie génique contenant le complexe correspondant comme ingrédient actif. En outre, la présente invention concerne le transporteur de gènes, un complexe de transport de gènes et une thérapie génique lutilisant. Il a été vérifié que le transporteur de gènes à base de polymère polydixylitol (PdXYP) de la présente invention a une vitesse de transport dacides nucléiques remarquablement plus élevée que les transporteurs daci
