Chromosome engineering: Prospects for gene therapy

Brenda Grimes, P. E. Warburton, C. J. Farr

Research output: Contribution to journalArticle

17 Citations (Scopus)

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.

Original languageEnglish (US)
Pages (from-to)713-718
Number of pages6
JournalGene Therapy
Volume9
Issue number11
DOIs
StatePublished - 2002
Externally publishedYes

Fingerprint

Human Artificial Chromosomes
Genetic Therapy
Chromosomes
Centromere
Artificial Chromosomes
Synthetic Genes
Satellite DNA
Hypoxanthine Phosphoribosyltransferase
Telomere
Italy
Medicine
Genome
Gene Expression

Keywords

  • Engineered chromosomes
  • Gene therapy vector

ASJC Scopus subject areas

  • Genetics

Cite this

Chromosome engineering : Prospects for gene therapy. / Grimes, Brenda; Warburton, P. E.; Farr, C. J.

In: Gene Therapy, Vol. 9, No. 11, 2002, p. 713-718.

Research output: Contribution to journalArticle

Grimes, Brenda ; Warburton, P. E. ; Farr, C. J. / Chromosome engineering : Prospects for gene therapy. In: Gene Therapy. 2002 ; Vol. 9, No. 11. pp. 713-718.
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