FIELD: molecular biology.
SUBSTANCE: gene therapy vector containing an expression cassette that contains a transgene encoding lysosome-associated membrane protein 2B (LAMP-2B) or a functional variant thereof is described, wherein said transgene is codon-optimized for expression in a human host cell, and wherein the sequence of said transgene has at least 95% identity to the sequence selected from SEQ ID NO: 3-5. A pharmaceutical composition for treating or preventing Danon disease, containing a gene therapy vector, is also described. A method of treating or preventing Danon disease or other autophagic disorder in a subject in need thereof, comprising administering to said subject a gene therapy vector or pharmaceutical composition, is presented.
EFFECT: claimed invention expands the arsenal of remedies for Danon's disease.
30 cl, 10 dwg, 4 ex
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