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Article

Development of allele-specific therapeutic siRNA for keratin 5 mutations in epidermolysis bullosa simplex.

The Journal of investigative dermatology - 1 Oct 2011

Atkinson Sarah D, McGilligan Victoria E, Liao Haihui, Szeverenyi Ildiko, Smith Frances J D, Moore C B Tara, McLean W H Irwin

Abstract excerpt

Epidermolysis bullosa simplex (EBS) is an incurable, inherited skin-blistering disorder predominantly caused by dominant-negative mutations in the genes encoding keratins K5 or K14. RNA interference, particularly in the form of small interfering RNA (siRNA), offers a potential therapy route for EBS and related keratin disorders by selectively silencing the mutant allele. Here, using a systemic screening system...

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PMID: 21716320
DOI: 10.1038/jid.2011.169

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