FIELD: biotechnology.
SUBSTANCE: invention relates to recombinant AAV expressing vectors, and can be used in medicine in treating a complement-related disease. Recombinant AAV vector with an expression cassette packed therein contains a nucleic acid encoding a human factor complement H (hfH) variant, functionally related to expression control sequences, which control its expression. Said version of hfH comprises a short consensus repeat (SCR) 1, 2, 3, 4, 19 and 20, and SCR 9, 10, 11, 12, 13, 14 and/or 15 are absent.
EFFECT: invention provides expressing a soluble variant of hfH protein preserving the complement regulation function.
17 cl, 37 dwg, 7 tbl
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Authors
Dates
2020-07-21—Published
2016-09-23—Filed