| abstract |
A synthetic RNA for use in the treatment of dystrophic epidermolysis bullosa, where the synthetic RNA encodes a gene-editing protein that targets a COL7 gene, where the synthetic RNA is suitable for in vivo administration to the keratinocytes of a subject , where the gene-editing protein edits the COL7 gene, and where the gene-editing protein comprises a DNA-binding domain and a nuclease domain, the gene-editing protein causing a single-stranded or double-stranded break in the gene COL7 from the patient's keratinocytes |