Review
Gene Therapy (2009) 16, 1180–1188; doi:10.1038/gt.2009.102; published online 27 August 2009
Progress and prospects: human artificial chromosomes
S Macnab1,2 and A Whitehouse1,2
- 1Institute of Molecular and Cellular Biology, Faculty of Biological Sciences, University of Leeds, Leeds, UK
- 2Astbury Centre for Structural Molecular Biology, University of Leeds, Leeds, UK
Correspondence: Dr A Whitehouse, Institute of Molecular and Cellular Biology, Faculty of Biological Sciences, University of Leeds, Leeds, LS2 9JT, UK. E-mail: a.whitehouse@leeds.ac.uk
Received 30 January 2009; Revised 25 July 2009; Accepted 28 July 2009; Published online 27 August 2009.
Abstract
Artificial chromosomes (ACs) are highly promising vectors for use in gene therapy applications. They are able to maintain expression of genomic-sized exogenous transgenes within target cells, without integrating into the host genome. Although these vectors have huge potential and benefits when compared against normal expression constructs, they are highly complex, technically challenging to construct and diffcult to deliver to target cells. This review focuses on the current progress in the field of ACs and discusses the recent advances in purification, construction, delivery and potential new molecular therapies.
Keywords:
HACs, BACs, YACs, amplicons, genomic transgenes, stem cells
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