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Medchemexpress LLC Rugonersen | 2591587-57-2 | 98.5% | 6530.30 | 5 MG
SDP

Supplier:  Medchemexpress LLC HY1481305MG

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Rugonersen is a locked-nucleic acid (LNA)-modified antisense oligonucleotide (ASO) that reduces ubiquitin-protein ligase E3A (UBE3A) silencing. It is used in research for Angelman syndrome (AS), a severe neurodevelopmental disorder caused by the loss of neuronal E3 ligase UBE3A.

  • Reduces ubiquitin-protein ligase E3A (UBE3A) silencing.
  • Utilized in Angelman syndrome (AS) research.
  • Features 98.5% purity.
  • Demonstrates in vitro nanomolar potency against UBE3A-ATS, UBE3A mRNA, and UBE3A protein upregulation.
  • Produces robust, long-lasting paternal reactivation of UBE3A mRNA/protein in cynomolgus monkeys.
  • Well-tolerated in monkey studies.
  • Available for research use only.

Catalog No. 50-003-63081


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