Allele Specific CRISPR/Cas9 Editing of Dominant Epidermolysis Bullosa Simplex in Human Epidermal Stem Cells

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Through the CRISPR/Cas9 system, scientists performed a specific cleavage only on the mutant allele and restored a normal cellular phenotype and a correct intermediate filament network, without affecting the epidermal stem cells, which play a crucial role in epidermal regeneration.
[Molecular Therapy]
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