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Mammalian Artificial Chromosomes and Clinical Applications for Genetic Modification of Stem Cells: An Overview

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Mammalian Chromosome Engineering

Part of the book series: Methods in Molecular Biology ((MIMB,volume 738))

Abstract

Modifying multipotent, self-renewing human stem cells with mammalian artificial chromosomes (MACs), present a promising clinical strategy for numerous diseases, especially ex vivo cell therapies that can benefit from constitutive or overexpression of therapeutic gene(s). MACs are nonintegrating, autonomously replicating, with the capacity to carry large cDNA or genomic sequences, which in turn enable potentially prolonged, safe, and regulated therapeutic transgene expression, and render MACs as attractive genetic vectors for “gene replacement” or for controlling differentiation pathways in progenitor cells. The status quo is that the most versatile target cell would be one that was pluripotent and self-renewing to address multiple disease target cell types, thus making multilineage stem cells, such as adult derived early progenitor cells and embryonic stem cells, as attractive universal host cells. We will describe the progress of MAC technologies, the subsequent modifications of stem cells, and discuss the establishment of MAC platform stem cell lines to facilitate proof-of-principle studies and preclinical development.

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Authors and Affiliations

  1. Institute of Genetics, Biological Research Center, Hungarian Academy of Sciences, Szeged, Hungary

    Robert L. Katona

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  1. Robert L. Katona
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  2. Sandra L. Vanderbyl
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  3. Carl F. Perez
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Correspondence to Robert L. Katona .

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Editors and Affiliations

  1. Biological Research Center, Institute of Genetics, Hungarian Academy of Sciences, Temesvári körút 62, Szeged, 6723, Hungary

    Gyula Hadlaczky

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Katona, R.L., Vanderbyl, S.L., Perez, C.F. (2011). Mammalian Artificial Chromosomes and Clinical Applications for Genetic Modification of Stem Cells: An Overview. In: Hadlaczky, G. (eds) Mammalian Chromosome Engineering. Methods in Molecular Biology, vol 738. Humana Press. https://doi.org/10.1007/978-1-61779-099-7_14

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  • DOI: https://doi.org/10.1007/978-1-61779-099-7_14

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  • Publisher Name: Humana Press

  • Print ISBN: 978-1-61779-098-0

  • Online ISBN: 978-1-61779-099-7

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