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SIRELRETIGENE SUBOPARVOVEC

UNII:45V2TAF3KB
Type:INGREDIENT SUBSTANCE
Formula:
Preferred Substance Name:SIRELRETIGENE SUBOPARVOVEC
GSRS Full Record:

  • 2395524-75-9
  • A NON-REPLICATING SELF-COMPLEMENTARY ADENO-ASSOCIATED VIRUS SEROTYPE 8 (SCAAV8) VECTOR, ENCODING HUMAN CELLULAR RETINALDEHYDE-BINDING PROTEIN 1 (RLBP1)
  • A RECOMBINANT NON-REPLICATING SELF-COMPLEMENTARY ADENO-ASSOCIATED VIRUS SEROTYPE 8 (SCAAV8) VECTOR, ENCODING HUMAN CELLULAR RETINALDEHYDE-BINDING PROTEIN 1 (RLBP1) UNDER THE CONTROL OF THE RETINALDEHYDE-BINDING PROTEIN 1 PROMOTER (PRLBP1) AND SV40 POLY ADENYLATION (POLYA) SEQUENCE. THE VECTOR GENOME IS A HEAD-TO-HEAD, SELF-COMPLEMENTARY DIMER, WITH THE VECTOR GENOME CASSETTE LOCATED 5' OF THE MUTATED INTERNAL INVERTED TERMINAL REPEAT (.DELTA.-ITR) IN A REVERSE COMPLEMENTARY ORIENTATION AND 3' OF THE .DELTA.-ITR IN A FORWARD ORIENTATION
  • DNA (SELF-COMPLEMENTARY ADENO-ASSOCIATED VIRUS 8 VECTOR SCAAV-PRLBP1-HRLBP1 HUMAN GENE RLBP1 PROMOTER PLUS HUMAN RETINALDEHYDE-BINDING PROTEIN RLBP1-SPECIFYING)
  • sirelretigene suboparvovec [INN]



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