CHUNG, Jong Hoon,GARG, Pankaj,PANDEY, Shambhavi,CHOUNG, Pill Hoon,CHO, Chong Su,KIM, Bog Hee
申请号：
EP15854007
公开号：
EP3214178A4
申请日:
2015.10.29
申请国别(地区):
EP
年份:
2018
代理人:
摘要：
The present invention relates to polydixylitol polymer based gene transporter (PdXYP) and a preparation method thereof. Further, the present invention relates to a nucleic acid delivery complex where the nucleic acids for treatment are conjugated to the gene transporter and a pharmaceutical composition for gene therapy including the complex as an active ingredient. In addition, the present invention relates to the gene transporter, gene delivery complex, and gene therapy using the gene transporter and gene delivery complex. It was confirmed that the PdXYP of the present invention has a considerably higher nucleic acid delivery rate than existing gene transporters, has almost no cytotoxicity in the conjugate when conjugated with DNA, also has very high in vivo transfection efficiency, and above all, especially has considerably high transfection efficiency for brain tissues, which has involved difficulty in gene therapy due to the blood brain barrier for a while. Accordingly, the gene transporter of the present invention can not only be used as experimental gene transporters, but can also be broadly used for various tissues in the body based on the nucleic acids for treatment to be conjugated in the field of gene therapy regarding various diseases.
