Therapeutic application of chimeric RNA/DNA oligonucleotide based gene therapy

Chimeric RNA/DNA oligonucleotides, or chimera, have emerged as a breakthrou gh technology for treating genetic disorders. Chimera have been shown to in duce correction of point mutations in several genetic disease models withou t utilising the viral vectors. Recent studies of chimera-based gene therapy in genetic disease models are reviewed. Chimera were delivered intravenous ly, intramuscularly, intradermally, or topically with or without vehicles. Correction of the mutation at genotypic and phenotypic levels was assessed using various methods. The gene correction frequency varied, ranging from 1 - 40%. The resulting phenotype changes lasted longer than one year in some studies. The most dramatic phenotypic change is the reduction of serum bil irubin level by 50% in the Gunn rat, a model for Crigler-Najjar syndrome. C himera based gene therapy has the potential to develop into powerful therap eutic modality for genetic diseases.