FIELD: biotechnology.
SUBSTANCE: invention relates to the field of biotechnology, specifically to genetic structures for expression of human mini-dystrophin; it can be used in medicine for the treatment of Duchenne muscular dystrophy (hereinafter – DMD) or another muscular disease related to DMD gene mutations. An expression cassette, a recombinant vector genome of adeno-associated virus (hereinafter – AAV), particles of a recombinant AAV (rAAV) for expression of human mini-dystrophin protein containing a nucleotide sequence encoding human mini-dystrophin protein with SEQ ID NO: 7, as well as production and application methods are proposed.
EFFECT: invention allows for an increase in the efficiency of therapy of muscular disease related to DMD gene mutations, as well as an increase in muscle mass and strength of an individual.
38 cl, 56 dwg, 13 tbl, 9 ex
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