CORRECTION OF THE MUTATION RESPONSIBLE FOR SICKLE-CELL-ANEMIA BY AN RNA-DNA OLIGONUCLEOTIDE

A chimeric oligonucleotide composed of DNA and modified RNA residues w as used to direct correction of the mutation in the hemoglobin beta(S) allele. After introduction of the chimeric molecule into lymphoblasto id cells homozygous for the beta(S) mutation, there was a detectable l evel of gene conversion of the mutant allele to the normal sequence. T he efficient and specific conversion directed by chimeric molecules ma y hold promise as a therapeutic method for the treatment of genetic di seases.