FIELD: biotechnology.
SUBSTANCE: invention relates to the field of biotechnology, in particular to a plasmid structure encoding the spCas9 protein under the control of the CAG promoter and expressing sgRNA against a fragment of the sequence 1 exon of the hprt1 gene under the control of the U6 promoter. A method for obtaining a line of mice for modeling the Lesch-Nyhan syndrome containing a deletion of p.Val8del in the mHprt1 gene using the above plasmid construction is also disclosed.
EFFECT: invention is effective for a detailed study of the pathogenesis and development of therapy for the Lesch-Nyhan syndrome.
2 cl, 2 dwg, 2 ex
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