Enhanced HDR-Mediated Correction of Heterozygous COL7A1 Mutations for Recessive Dystrophic Epidermolysis Bullosa

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Scientists developed a highly efficient and versatile gene editing strategy for recessive dystrophic epidermolysis bullosa that incorporated the small molecule inhibitor M3814 to enhance homology-directed repair (HDR).
[Molecular Therapy – Nucleic Acids]
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