Article
Gene therapy for autosomal dominant disorders of keratin.
The journal of investigative dermatology. Symposium proceedings - 1 Oct 2005
Lewin Alfred S, Glazer Peter M, Milstone Leonard M
Abstract excerpt
Dominant mutations that interfere with the assembly of keratin filaments cause painful and disfiguring epidermal diseases like pachyonychia congenita and epidermolysis bullosa simplex. Genetic therapies for such diseases must either suppress the production of the toxic proteins or correct the genetic defect in the chromosome. Because epidermal skin cells may be genetically modified in tissue culture or in situ,...
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