FIELD: biotechnology; medicine.
SUBSTANCE: combination of the treatment of congenital adrenal cortex dysfunction is described. The combination contains the following: an expression vector containing the sequence of Cas9 gene of Streptococcus pyogenes, as well as an expression vector containing the sequence of hCYP21A2 gene. The expression vectors are adeno-associated virus (AAV) viral particles. Also the following is disclosed: a method of the treatment of congenital dysfunction of the adrenal cortex, including the introduction into the body of a mammal of a combination according to claim 1 in an effective amount.
EFFECT: invention expands the range of medicinal products for the treatment of congenital dysfunction of the adrenal cortex.
10 cl, 9 dwg, 4 tbl, 5 ex
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