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Session IV – Chromosome Engineering

Chromosome engineering: prospects for gene therapy

Gene Therapy volume 9, pages 713–718 (2002)Cite this article

Abstract

Recent advances in chromosome engineering and the potential for downstream applications in gene therapy were presented at the Artificial Chromosome Session of Genome Medicine: Gene Therapy for the Millennium in Rome, Italy in September 2001. This session concentrated primarily on the structure and function of human centromeres and the ongoing challenge of equipping human artificial chromosomes (HACs) with centromeres to ensure their mitotic stability. Advances in the ‘bottom up’ construction of HACs included the transfer into HT1080 cells of circular PACs containing alpha satellite DNA, and the correction of HPRT deficiency in cells using HACs. Advances in the ‘top down’ construction of HACs using telomere associated chromosome fragmentation in DT40 cells included the formation of HACs that are less than a megabase in size and transfer of HACs through the mouse germline. Significant progress has also been made in the use of human minichromosomes for stable trans-gene expression. While many obstacles remain towards the use of HACs for gene therapy, this session provided an optimistic outlook for future success.

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Acknowledgements

We would like to thank meeting participants Hunt Willard, Bala Balakumaran, Katie Rudd and Roger Slee for valuable discussions.

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Authors and Affiliations

  1. Department of Genetics, School of Medicine, Case Western Reserve University and University Hospitals of Cleveland, Cleveland, OH, USA

    B R Grimes

  2. Mount Sinai School of Medicine, New York, NY, USA

    P E Warburton

  3. Department of Genetics, University of Cambridge, Cambridge, UK

    C J Farr

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  1. B R Grimes
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  2. P E Warburton
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  3. C J Farr
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Grimes, B., Warburton, P. & Farr, C. Chromosome engineering: prospects for gene therapy. Gene Ther 9, 713–718 (2002). https://doi.org/10.1038/sj.gt.3301763

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