FIELD: biotechnology.
SUBSTANCE: invention relates to biotechnology and namely to the construction of a nucleic acid for gene therapy that contains a variant ATP7B in which the N-terminal areas of HMA1, HMA2, HMA3 and HMA4 are absent, and the N-terminal areas of HMA5 and HMA6 are present. Also disclosed are an expression vector, a host cell, a virion, a kit, a pharmaceutical composition and their use for treating a condition caused by deficiency or dysfunction of copper-transporting ATPase 2. The invention also relates to a method for producing a virion and a method for treating a condition in a patient caused by deficiency or dysfunction of copper-transporting ATPase.
EFFECT: invention is used for treatment of conditions associated with deficiency or dysfunction of copper-transporting ATPase 2, in particular for treatment of Wilson's disease.
28 cl, 14 dwg, 8 ex
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