FIELD: biotechnology.
SUBSTANCE: present invention relates to biotechnology, specifically to mini-protein USH2A, which, with a small size, which enables delivery as a part of viral vectors, preserves domains of the full-length protein LamGL, LamNT, EGF Lam, FN3 and PDZ-binding motif, which are necessary for the functioning of the USH2A protein. Also disclosed is a nucleic acid coding a mini-protein USH2A, an expression vector and the use of said vector in a gene therapy of type II Usher syndrome.
EFFECT: invention is effective for stable and high expression of USH2A mini-protein.
13 cl, 11 dwg, 8 tbl, 6 ex
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