FIELD: biotechnology.
SUBSTANCE: invention relates to a nucleic acid construct containing a 5'-inverted terminal repeat (ITR) sequence, a promoter sequence, a nucleic acid sequence encoding truncated human copper-transporting ATPase 2 (ATP7B), a 3'-ITR sequence, and also to the composition containing it.
EFFECT: invention is effective for the treatment of Wilson's disease.
20 cl, 12 dwg, 6 ex
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