FIELD: biotechnology.
SUBSTANCE: adeno-associated virus (hereinafter – AAV) containing capsid protein, a composition for the treatment of a muscle-related disease or disorder, a method for the delivery of a transgene to a muscle cell, a method for the treatment of a subject suffering from a muscle-related disorder or disease, an expression vector, a cell for the expression of AAV capsid protein, the use of AAV in the manufacture of a drug for the treatment of a muscle-related disorder or diseases; cDNA containing a nucleotide sequence encoding capsid protein, a method for targeting a transgene to a muscle cell, and a method for the production of an adeno-associated virus (AAV) are presented.
EFFECT: invention allows for the use of an adeno-associated virus (AAV) in gene therapy methods.
72 cl, 10 tbl, 3 ex
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Authors
Dates
2022-09-06—Published
2017-07-25—Filed