Eteplirsen

Names

[ Name ]:
Eteplirsen

[Synonym ]:
AVI-4658
Exondys 51
(P-deoxy-P-(dimethylamino)](2',3'-dideoxy-2',3'-imino-2',3'-seco)(2'a→5')(C-m5U-C-C-A-A-C-A-m5U-C-A-A-G-G-A-A-G-A-m5U-G-G-C-A-m5U-m5U-m5U-C-m5U-A-G),5'-(P-(4-((2-(2-(2-hydroxyethoxy)ethoxy)ethoxy)carbonyl)-1-piperazinyl)-N,N-dimethylphosphonamidate) RNA

Biological Activity

[Description]:

Eteplirsen (AVI 4658) is a synthetic antisense oligonucleotide. Eteplirsen can be used for Duchenne muscular dystrophy research[1].

[Related Catalog]:

Signaling Pathways >> Others >> Others

Research Areas >> Metabolic Disease

[In Vitro]

Eteplirsen acts to promote dystrophin production by restoring the translational reading frame of Duchenne muscular dystrophy (DMD) through specific skipping of exon 51 in defective gene variants[1].

[References]

[1]. Kenji Rowel Q Lim, et al. Eteplirsen in the treatment of Duchenne muscular dystrophy. Drug Des Devel Ther. 2017 Feb 28;11:533-545.

Chemical & Physical Properties

[ Molecular Formula ]:
C₃₆₄H₅₆₉N₁₇₇O₁₂₂P₃₀

[ Molecular Weight ]:
10305.74000

[ Exact Mass ]:
10300.59000

Related Compounds

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