FIELD: biotechnology.
SUBSTANCE: invention relates to the field of biotechnology, in particular to a composition for the treatment of Leber congenital amaurosis related to a deep intron mutation in CEP290 gene of an individual based on CRISPR-Cas system, where Cas protein splits Cas expression cassette thereby reducing expression of Cas protein, as well as to a method for the treatment of Leber congenital amaurosis using it. A kit containing the above-mentioned composition, and a viral particle containing the above-mentioned CRISPR-Cas system are also presented.
EFFECT: invention provides for a favorable clinical result, when treating Leber congenital amaurosis.
13 cl, 8 dwg, 3 tbl, 5 ex
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