Site-specific genome editing for correction of induced pluripotent stem cells derived from dominant dystrophic epidermolysis bullosa

Significance Some inherited autosomal dominant disorders are caused by dominant negative mutations whose gene product adversely affects the normal gene product expressed from the other allele. Gene editing with engineered site-specific endonucleases leads to a double-strand break, which promotes nonhomologous end-joining (NHEJ). NHEJ is error-prone and conducive to the generation of frameshift mutations, leading to intentional knockout of a...