alexa Toward Gene and Cell Therapies Employing Human Artifici
ISSN: 2168-9849

Cloning & Transgenesis
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Review Article

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

Narumi Uno1, Yasuhiro Kazuki1,2 and Mitsuo Oshimura1,2*
1Department of Biomedical Science, Institute of Regenerative Medicine and Biofunction, Graduate School of Medical Science, Japan
2Chromosome Engineering Research Center (CERC), Tottori University, Nishicho 86, Yonago, Japan
Corresponding Author : Mitsuo Oshimura
Department of Biomedical Science
Institute of Regenerative Medicine and Biofunction
Graduate School of Medical Science
Tottori University, Nishicho 86, Yonago, Japan
Tel: +81-859-38-6211
Fax: +81-859-38-6210
E-mail: [email protected]
Received Janauray 12, 2014; Accepted February 14, 2014; Published February 16, 2014
Citation: Uno N, Kazuki Y, Oshimura M (2014) Toward Gene and Cell Therapies Employing Human Artificial Chromosomes in Conjunction with Stem Cells. Clon Transgen 3:122. doi:10.4172/2168-9849.1000122
Copyright: © 2014 Uno N, et al.. This is an open-access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.


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.

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