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BRINRETIGENE VESGEDPARVOVEC

UNII:4G7DT7PP3L
Formula:
Preferred Substance Name:BRINRETIGENE VESGEDPARVOVEC

  • 2768756-11-0
  • BRINRETIGENE VESGEDPARVOVEC [INN]
  • DNA (RECOMBINANT ADENO-ASSOCIATED VIRUS VECTOR EDIT-101 HUMAN U6 PROMOTER PLUS NEPHROCYSTIN 6 INTRON 26-SPECIFIC CEP290-323 GUIDE RNA GENE PLUS HUMAN U6 PROMOTER PLUS NEPHROCYSTIN 6 INTRON 26-SPECIFIC CEP290-64 GUIDE RNA GENE PLUS HUMAN RHODOPSIN KINASE PROMOTER PLUS SIMIAN VIRUS 40 NUCLEAR LOCALIZATION SIGNAL PEPTIDE FUSION PROTEIN WITH STAPHYLOCOCCUS AUREUS CRISPR-ASSOCIATED ENDODEOXYRIBONUCLEASE CAS9 FUSION PROTEIN WITH SIMIAN VIRUS 40 NUCLEAR LOCALIZATION SIGNAL PEPTIDE GENE PLUS SYNTHETIC POLYADENYLATION SIGNAL PLUS ADENO-ASSOCIATED VIRUS 2 INVERTED TERMINAL REPEAT FLANKS-SPECIFYING)
  • EDIT-101
  • 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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