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

@article{Atkinson2011DevelopmentOA,
  title={Development of allele-specific therapeutic siRNA for keratin 5 mutations in epidermolysis bullosa simplex.},
  author={Sarah D. Atkinson and Victoria McGilligan and Haihui Liao and Ildik{\'o} Szever{\'e}nyi and Frances J. D. Smith and Charles T. Moore and W. H. Irwin McLean},
  journal={The Journal of investigative dermatology},
  year={2011},
  volume={131 10},
  pages={
          2079-86
        }
}
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 based on a luciferase reporter gene assay, we have developed mutant-specific siRNAs… CONTINUE READING

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