Article
Functional improvement of mutant keratin cells on addition of desmin: an alternative approach to gene therapy for dominant diseases.
Gene therapy - 1 Aug 2004
D'Alessandro M, Morley S M, Ogden P H, Liovic M, Porter R M, Lane E B
Abstract excerpt
A major challenge to the concept of gene therapy for dominant disorders is the silencing or repairing of the mutant allele. Supplementation therapy is an alternative approach that aims to bypass the defective gene by inducing the expression of another gene, with similar function but not susceptible to the disrupting effect of the mutant one. Epidermolysis bullosa simplex (EBS) is a genetic skin fragility disorder...
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