Targeted gene conversion in a mammalian CD34+-enriched cell population using a chimeric RNA/DNA oligonucleotide

Gene conversion of genetically inherited point mutations is a fundamental methodology for treating a variety of diseases. We tested the feasibility of a new approach using an RNA/DNA chimeric oligonucleotide. The beta-globin gene was targeted at the point mutation causing sickle cell anemia. The chi...

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Veröffentlicht in:Journal of molecular medicine (Berlin, Germany) Germany), 1997-11, Vol.75 (11-12), p.829-835
Hauptverfasser: XIANG, Y, COLE-STRAUSS, A, YOON, K, GRYN, J, KMIEC, E. B
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Sprache:eng
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Zusammenfassung:Gene conversion of genetically inherited point mutations is a fundamental methodology for treating a variety of diseases. We tested the feasibility of a new approach using an RNA/DNA chimeric oligonucleotide. The beta-globin gene was targeted at the point mutation causing sickle cell anemia. The chimera is designed to convert an A residue to a T after creating a mismatched basepair. In a CD34+-enriched population of normal cells a 5-11% conversion rate was measured using restriction enzyme polymorphism and direct DNA sequence analyses. The closely related delta-globin gene sequence appeared unchanged despite successful conversion at the beta-globin locus.
ISSN:0946-2716
1432-1440
DOI:10.1007/s001090050172