Article
SiRNA-mediated selective inhibition of mutant keratin mRNAs responsible for the skin disorder pachyonychia congenita.
Annals of the New York Academy of Sciences - 1 Oct 2006
Hickerson Robyn P, Smith Frances J D, McLean W H Irwin, Landthaler Markus, Leube Rudolf E, Kaspar Roger L
Abstract excerpt
RNA interference offers a novel approach for treating genetic disorders including the rare monogenic skin disorder pachyonychia congenita (PC). PC is caused by mutations in keratin 6a (K6a), K6b, K16, and K17 genes, including small deletions and single nucleotide changes. Transfection experiments of a fusion gene consisting of K6a and a yellow fluorescent reporter (YFP) resulted in normal keratin filament...
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