次世代遺伝子治療薬の開発基盤研究

抄録

Successful gene therapy depends largely on vectors that can efficiently deliver the therapeutic genes into the target tissues and cells. Recombinant adenovirus (Ad) vectors continue to be the preferred vectors for gene therapy because they can easily be grown to high titers and can efficiently transfer genes into both dividing and nondividing cells. However, there are some limitations such as the time-consuming and labor-intensive procedures for vector construction, coxsackievirus-adenovirus receptor (CAR)-dependent gene transfer, immunologic side effects, lack of tissue specificity, lack of regulation of gene expression, etc. In this paper, I review our approach to the development of advanced recombinant Ad vectors. The next generation of Ad vectors have not only become promising vectors for gene therapy but also important tools for gene transfer into mammalian cells.