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Chromosome engineering: prospects for gene therapy.

B R Grimes¹, P E Warburton, C J Farr

¹Department of Genetics, School of Medicine, Case Western Reserve University and University Hospital of Cleveland, Cleveland, OH 44106, USA.

|May 29, 2002

Summary

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Researchers are advancing human artificial chromosome (HAC) technology for gene therapy. Progress in centromere function and construction methods offers an optimistic outlook for stable gene expression and therapeutic applications.

Area of Science:

Genetics
Molecular Biology
Gene Therapy

Background:

The development of human artificial chromosomes (HACs) is crucial for gene therapy.
Ensuring mitotic stability of HACs through functional centromeres remains a key challenge.

Purpose of the Study:

To present recent advances in HAC construction and their potential for gene therapy.
To discuss the structure and function of human centromeres in the context of HACs.

Main Methods:

'Bottom up' construction: Transfer of circular PACs with alpha satellite DNA into HT1080 cells.
'Top down' construction: Telomere-associated chromosome fragmentation in DT40 cells to create small HACs.
Utilizing HACs for gene correction (HPRT deficiency) and stable transgene expression.

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Main Results:

Demonstrated successful transfer and mitotic stability of HACs in cell lines.
Created HACs smaller than one megabase and achieved germline transmission in mice.
Showcased stable transgene expression using human minichromosomes.

Conclusions:

Significant progress has been made in HAC engineering for gene therapy applications.
While challenges persist, the outlook for HAC-based gene therapy is optimistic.