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Science 6 September 1996:
Vol. 273. no. 5280, pp. 1386 - 1389
DOI: 10.1126/science.273.5280.1386
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Reports

Correction of the Mutation Responsible for Sickle Cell Anemia by an RNA-DNA Oligonucleotide

Allyson Cole-Strauss, * Kyonggeun Yoon, * Yufei Xiang, Bruce C. Byrne, Michael C. Rice, Jeff Gryn, William K. Holloman, Eric B. Kmiec dagger

A chimeric oligonucleotide composed of DNA and modified RNA residues was used to direct correction of the mutation in the hemoglobin beta S allele. After introduction of the chimeric molecule into lymphoblastoid cells homozygous for the beta S mutation, there was a detectable level of gene conversion of the mutant allele to the normal sequence. The efficient and specific conversion directed by chimeric molecules may hold promise as a therapeutic method for the treatment of genetic diseases.

A. Cole-Strauss, K. Yoon, Y. Xiang, M. C. Rice, E. B. Kmiec, Department of Pharmacology, Kimmel Cancer Center, Thomas Jefferson University, 233 South 10th Street, Philadelphia, PA 19107, USA.
B. C. Byrne and J. Gryn, Division of Hematology-Oncology, Department of Medicine, Cooper Hospital University Medical Center, Camden, NJ 08103, USA.
W. K. Holloman, Department of Microbiology, Cornell University School of Medicine, 1300 York Avenue, New York, NY, USA.
*   These authors contributed equally to this work.

dagger    To whom correspondence should be addressed.


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