Бринретиген весгедпарвовек

Rec. INN (наименование, зарегистрированное ВОЗ)

2768756-11-0

recombinant, non-replicating adeno-associated virus serotype 5 (rAAV5) vector encoding (1) codon- optimized Staphylococcus aureus clustered regularly interspaced short palindromic repeats (CRISPR)- associated protein 9 (Cas9) flanked by simian virus 40 (SV40) nuclear localisation signal peptide sequences and (2) two guide RNAs (gRNA-323 and gRNA-64) targeting intron 26 of the human centrosomal protein 290 (CEP290, nephrocystin-6) gene. Expression of the CRISPR Cas9 gene is under control of the human G protein-coupled receptor kinase 1 (GRK1, rhodopsin kinase) promoter along with an SV40 splice donor/splice acceptor site, a Kozak sequence and a small synthetic polyadenylation signal. Expression of the two gRNAs is each under control of a human small nuclear RNA U6 polymerase III promoter. The vector genome is flanked by adeno-associated virus 2 (AAV2) inverted terminal repeats (ITRs). Expression of the gene editing components is intended to remove or invert the mutation-containing intronic region of the CEP290 gene (IVS26), and to restore functional CEP290 protein levels

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