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Science. 2000 Nov 17;290(5495):1308-9.

Genomics and gene therapy. Artificial chromosomes coming to life.

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  • 1Department of Genetics and Center for Human Genetics at Case Western Reserve University and the Research Institute of Universi Hospitals of Cleveland, Cleveland, OH 44106, USA.


One of the biggest obstacles to gene therapy is the delivery of the therapeutic gene to the target tissue so that it is appropriately expressed. In his Perspective, Willard looks at the potential advantages of using a human artificial chromosome to maintain expression of a therapeutic gene and discusses some of the hurdles yet to be overcome before this gene delivery system can be tried out in the clinic.

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