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ROVOCTOCOGENE DURPARVOVEC

UNII:63QD0BX9B4
Type:INGREDIENT SUBSTANCE
Formula:
Preferred Substance Name:ROVOCTOCOGENE DURPARVOVEC
GSRS Full Record:

  • 2163074-26-6
  • A RECOMBINANT INTRONLESS NON-REPLICATING ADENO-ASSOCIATED VIRUS 2 VECTOR (AAV2) WITH A BIO-ENGINEERED CAPSID DERIVED BY DNA SHUFFLING OF 10 AAV CAPSID SEQUENCES (AAV1, 2, 3B, 4, 5, 6 8, 9, AVIAN AND BOVINE AAV) FOLLOWED BY IN VIVO SELECTION IN FAH//RAG2//IL2RG/ (FRG) MICE PARTIALLY REPOPULATED WITH HUMAN HEPATOCYTES. THE VECTOR HAS AAV2 GENOMIC INVERTED TERMINAL REPEATS (ITR) AND ENCODES A HUMAN B-DOMAIN-DELETED BLOOD COAGULATION FACTOR VIII (HF8, FACTOR VIII, FVIII) UNDER THE CONTROL OF A MODIFIED TRANSTHYRETIN (TTRM) PROMOTER.
  • rovoctocogene durparvovec [INN]



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