Article
PNA as a potential modulator of COL7A1 gene expression in dominant dystrophic epidermolysis bullosa: a physico-chemical study.
Molecular bioSystems - 1 Dec 2013
Amato Jussara, Stellato Marco Ignazio, Pizzo Elio, Petraccone Luigi, Oliviero Giorgia, Borbone Nicola, Piccialli Gennaro, Orecchia Angela, Bellei Barbara, Castiglia Daniele, Giancola Concetta
Abstract excerpt
Dominant diseases are single gene disorders occurring in the heterozygous state. The mutated allele exerts a dominant effect because it produces an abnormal polypeptide that interferes with the function of the normal allele product. Peptide Nucleic Acids (PNAs) offer a route for a potential therapy for dominant diseases by selectively silencing the allele carrying the dominant mutation. Here, we have synthesized...
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