クローニングと遺伝子導入
オープンアクセス

概要

Toward Gene and Cell Therapies Employing Human Artificial Chromosomes in Conjunction with Stem Cells

Narumi Uno Yasuhiro Kazuki and Mitsuo Oshimura

Various Stem cells including induced pluripotent stem cells (iPSCs) are expected to contribute to the development of regenerative medicines. Human artificial chromosome (HAC) vectors have no limits with respect to the size of inserts, and are independently maintained from host chromosomes. HACs could be promising vectors that will contribute to gene and cell therapy in the future. Various applications and methods have been developed using HAC vectors; however, in this review we introduce current research focused towards gene and cell therapy of Duchenne muscular dystrophy and hemophilia A using stem cells. In addition, HAC vectors to generate iPSCs, with a safeguard system to eliminate tumorigenic cells are proposed.