Targeted correction of the point mutations of β-thalassemia and targeted mutagenesis of the nucleotide associated with HPFH by RNA/DNA oligonucleotides: potential …

ZH Li, DP Liu, WX Yin, ZC Guo, CC Liang - Blood Cells, Molecules, and …, 2001 - Elsevier
An RNA/DNA chimeric oligonucleotide was found to be effective in the targeted correction of
point mutations in Escherichia coli, plant, and mammalian genomes. This strategy, named
chimeraplasty, has the potential for gene therapy of many genetic diseases caused by point
mutations. β-Thalassemia is a very common human genetic disease and in most cases it is
caused by point mutations. To test whether the chimeraplasty can be used to correct the
point mutations responsible for β-thalassemia, we introduced one mutated β-globin gene …