FIELD: biotechnology; medicine.
SUBSTANCE: invention relates to biotechnology and medicine and is a method of treating phenylketonuria (PKU) in an individual in need thereof. Invention also relates to an expression cassette, a vector, including rAAV-based, as well as a kit for providing expression of a PAH polypeptide variant in an individual in need thereof.
EFFECT: invention provides clinical effectiveness in phenylketonuria.
17 cl, 17 dwg, 3 tbl, 7 ex
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