FIELD: biotechnology.
SUBSTANCE: present invention relates to biotechnology, specifically to editing of genes, and can be used in medicine. Method for treating dystrophic bullous epidermolysis involves delivering a patient's keratinocytes by introducing into the epidermis a synthetic RNA coding a protein-editing gene which targets the COL7 gene and causes a double-strand break in the COL7 gene, and a matrix delivery for the COL7 repair to the patient, for editing the COL7 gene. Also presented is a method wherein a synthetic RNA coding a gene-editing protein which is aimed at the COL7 gene is delivered into the patient's keratinocytes by injection into the epidermis, and inducing a single- or double-strand break in the COL7 gene to remove a mutation which is at least partially responsible for the phenotype of the disease.
EFFECT: invention provides improving one or more symptoms of dystrophic bullous epidermolysis in a patient.
7 cl, 18 dwg, 4 tbl, 56 ex
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