FIELD: biotechnology.
SUBSTANCE: invention refers to a "zinc fingers" protein of non-natural origin that binds to the Htt gene, which can be used in medicine. The specified "zinc fingers" protein, as well as fusion protein that includes the specified protein functionally associated with a nuclease domain, a polynucleotide, encoding the specified protein and the specified fusion protein, a host cell for obtaining of the specified protein and the specified fusion protein, a pharmaceutical composition for Htt gene expression suppression in the cell are obtained. The invention also provides a method for Htt gene expression suppression, a method for Huntington's disease treatment.
EFFECT: invention effectively suppresses the Htt gene expression, which allows successful treatment of Huntington's disease.
24 cl, 15 dwg, 7 tbl, 20 ex
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Authors
Dates
2017-12-20—Published
2013-02-28—Filed