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Human artificial chromosome vectors meet stem cells

New prospects for gene delivery

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Abstract

The recent emergence of stem cell-based tissue engineering has now opened up new venues for gene therapy. The task now is to develop safe and effective vectors that can deliver therapeutic genes into specific stem cell lines and maintain long-term regulated expression of these genes. Human artificial chromosomes (HACs) possess several characteristics that require gene therapy vectors, including a stable episomal maintenance, and the capacity for large gene inserts. HACs can also carry genomic loci with regulatory elements, thus allowing for the expression of transgenes in a genetic environment similar to the chromosome. Currently, HACs are constructed by a two prone approaches. Using a top-down strategy, HACs can be generated from fragmenting endogenous chromosomes. By a bottom-up strategy, HACs can be created de novo from cloned chromosomal components using chromosome engineering. This review describes the current advances in developing HACs, with the main focus on their applications and potential value in gene delivery, such as HAC-mediated gene expression in embryonic, adult stem cells, and transgenic animals.

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Authors and Affiliations

  1. Department of Biomedical Science, Institute of Regenerative Medicine and Biofunction Graduate School of Medical Science, Tottori University, 86 Nishicho, Yonago, 683-8503, Tottori, Japan

    Xianying Ren, Candice Ginn T. Tahimic, Akihiro Kurimasa & Mitsuo Oshimura

  2. Department of Human Genome Sciences, Graduate School of Medical Science, Tottori University, 86 Nishicho, Yonago, 683-8503, Tottori, Japan

    Motonobu Katoh & Toshiaki Inoue

  3. Chromocenter Inc., 8-14-39 Yonehara, Yonago, 683-0804, Tottori, Japan

    Mitsuo Oshimura

Authors
  1. Xianying Ren
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  2. Candice Ginn T. Tahimic
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  3. Motonobu Katoh
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  4. Akihiro Kurimasa
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  5. Toshiaki Inoue
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Correspondence to Mitsuo Oshimura.

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Ren, X., Tahimic, C.G.T., Katoh, M. et al. Human artificial chromosome vectors meet stem cells. Stem Cell Rev 2, 43–50 (2006). https://doi.org/10.1007/s12015-006-0008-9

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