Seoul National University

Snu Rdb Foundation

Shambhavi Pandey

Pill Hoon Choung

Jong Hoon Chung

Pankaj Garg

Chong Su Cho

Bog Hee Kim

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.

Polyol-based osmotic polydixylitol polymer gene transporter and use thereof

The present invention relates to a vitamin B6-coupled poly(ester amine) (VBPEA) as a gene carrier and a method for preparing the gene carrier. Moreover, the present invention relates to a gene delivery complex comprising a therapeutic gene coupled to the gene carrier and a pharmaceutical formulation for gene therapy, which comprises the gene delivery complex as an active ingredient. In addition, the present invention relates to gene therapy utilizing the gene carrier, the gene delivery complex or the pharmaceutical formulation. The VBPEA of the invention has a significantly high gene delivery rate compared to existing gene carriers and a complex of the VBPEA with DNA has little or no cytotoxicity and shows a very high in vivo transfection efficiency. In addition, a complex of the VBPEA with siRNA shows high gene silencing efficiency and can induce a high rate of cell death and the inhibition of cell proliferation in cancer cells, suggesting that it can be used for anticancer gene therapy. Thus, the gene carrier VBPEA of the invention can be used as an experimental gene carrier and can also be widely used in gene therapy against various diseases depending on the kind of therapeutic gene.

Vitamin B6-coupled poly(ester amine) as gene carrier and application in cancer gene therapy

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Seoul, South Korea

Shambhavi Pandey