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FORDADISTROGENE MOVAPARVOVEC

UNII:H4T32JIH01
Type:INGREDIENT SUBSTANCE
Formula:
Preferred Substance Name:FORDADISTROGENE MOVAPARVOVEC
GSRS Full Record:
H4T32JIH01
i

  • 2248111-37-5
  • AAV9.HCK.OPTI-DYS.DELTA.3978
  • DNA (RECOMBINANT ADENO-ASSOCIATED VIRUS 9 VECTOR PF-06939926 HUMAN MUSCLE-SPECIFIC ENHANCER/PROMOTER PLUS HUMAN MINI-DYSTROPHIN GENE PLUS ADENO-ASSOCIATED VIRUS 9 INVERTED TERMINAL REPEAT FLANKS)
  • FORDADISTROGENE MOVAPARVOVEC [USAN]
  • Fordadistrogene movaparvovec [WHO-DD]
  • PF-06939926
  • PF-06939926 IS A RECOMBINANT AAV VECTOR CONTAINING THE AAV SEROTYPE 9 (AAV9) CAPSID PACKAGING A MINIATURIZED CODON-OPTIMIZED VERSION OF THE DMD GENE(CALLED OPTI-DYS.DELTA.3978) WHICH ENCODES THE DOMAINS MINIMALLY REQUIRED FOR FUNCTIONALITY OF THE DYSTROPHIN PROTEIN (MINI-DYSTROPHIN). A SYNTHETIC POLYADENYLATION (SPOLYA) SEQUENCE FOLLOWS THE OPTI-DYS.DELTA.3978 GENE. CARDIAC AND SKELETAL MUSCLE-SPECIFIC TRANSGENE EXPRESSION IS MEDIATED BY A SYNTHETIC ENHANCER AND PROMOTER. THE EXPRESSION CONSTRUCT IS FLANKED BY THE AAV2 INVERTED TERMINAL REPEATS (ITRS). THE 3978 BASEPAIR MINI-DYSTROPHIN OPEN READING FRAME ENCODES THE N-TERMINUSHINGE REGIONS H1, H3 AND H4SPECTRIN-LIKE REPEATS R1, R2 AND R22-24AND THE C-TERMINAL CYSTEINE-RICH REGION OF THE DYSTROPHIN PROTEIN.
  • fordadistrogene movaparvovec [INN]




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