FIELD: biotechnology.
SUBSTANCE: invention relates to the field of biotechnology, in particular to a method for in vivo assessment of therapeutic efficiency of a remedy for use in the treatment of complement-related nephropathy. The method provides for administration of a remedy to a rodent, in the genome of which there is a substitution by endogenous locus C3 of a rodent of a rodent gene sequence containing C3 gene exon with a nucleic acid sequence containing at least one human C3 gene exon to form a modified C3 gene. A method is also disclosed for identification of a remedy that suppresses a symptom of complement-related nephropathy in a rodent, in the genome of which there is a substitution by endogenous locus C3 of a rodent of a rodent gene sequence encoding C3 gene exon with a nucleic acid sequence containing at least one human C3 gene exon to form a modified C3 gene.
EFFECT: invention is effective for screening of candidate therapeutic remedies or compounds for the treatment of complement-related nephropathies and liver fibrosis.
17 cl, 31 dwg, 9 ex
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Authors
Dates
2022-05-23—Published
2018-02-26—Filed