Abstract
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 β-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 δ-globin gene sequence appeared unchanged despite successful conversion at the β-globin locus.
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Received: 21 January 1997 / Accepted: 18 March 1997
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Xiang, Y., Cole-Strauss, A., Yoon, K. et al. Targeted gene conversion in a mammalian CD34+-enriched cell population using a chimeric RNA/DNA oligonucleotide. J Mol Med 75, 829–835 (1997). https://doi.org/10.1007/s001090050172
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DOI: https://doi.org/10.1007/s001090050172