FIELD: biotechnology.
SUBSTANCE: invention relates to the field of biotechnology, in particular to a vector based on adeno-associated virus (AAV) of serotype 9, containing an expression cassette containing polynucleotide encoding lysosome-associated membrane protein 2B (LAMP-2B), functionally connected to a hybrid promotor containing chicken β-actin promotor and CMV enhancer (CAG promotor).
EFFECT: invention is effective for the treatment of Danon’s disease.
14 cl, 11 dwg, 8 ex
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