FIELD: biotechnology.
SUBSTANCE: invention relates to the field of biotechnology, in particular to a complex for editing the genome of a mouse egg cell based on CRISPR/Cas9, consisting of mRNA of the S. using. Also disclosed is a mouse strain with a humanized nucleotide sequence of exon 6 of the Gnao1 gene.
EFFECT: invention is effective for in vivo testing of the specificity of RNA therapy drugs for patients with variant c.607 G>A GNAO1 encephalopathy.
4 cl, 6 dwg, 4 ex
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| GENOMIC ENGINEERING | 2014 |
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