1173755-55-9
Chemical Structure
Eteplirsen
Synonym(s): AVI 4658
- CAS No.: 1173755-55-9
- Formula:C364H569N177O122P30
- Molecular Weight:10305.74
SMILES: O=C1NC(N)=NC2=C1N=CN2[C@H]3CNC[C@H](O3)COP(N4C[C@H](O[C@@H](N5C(N=CN=C6N)=C6N=C5)C4)COP(N7C[C@@H](COP(N8C[C@@H](COP(N9C[C@@H](COP(N%10C[C@@H](COP(N%11C[C@@H](COP(N%12C[C@@H](COP(N%13C[C@@H](COP(N%14C[C@@H](COP(N%15C[C@@H](COP(N%16C[C@@H](COP(N%17C[C@@H](COP(N%18C[C@@H](COP(N%19C[C@@H](COP(N%20C[C@@H](COP(N%21C[C@@H](COP(N%22C[C@@H](COP(N%23C[C@@H](COP(N%24C[C@@H](COP(N%25C[C@@H](COP(N%26C[C@@H](COP(N%27C[C@@H](COP(N%28C[C@@H](COP(N%29C[C@@H](COP(N%30C[C@@H](COP(N%31C[C@@H](COP(N%32C[C@@H](COP(N%33C[C@@H](COP(N%34C[C@@H](COP(N%35CCN(C(OCCOCCOCCO)=O)CC%35)(N(C)C)=O)O[C@@H](N%36C(N=C(N)C=C%36)=O)C%34)(N(C)C)=O)O[C@@H](N%37C(NC(C(C)=C%37)=O)=O)C%33)(N(C)C)=O)O[C@@H](N%38C(N=C(N)C=C%38)=O)C%32)(N(C)C)=O)O[C@@H](N%39C(N=C(N)C=C%39)=O)C%31)(N(C)C)=O)O[C@@H](N%40C(N=CN=C%41N)=C%41N=C%40)C%30)(N(C)C)=O)O[C@@H](N%42C(N=CN=C%43N)=C%43N=C%42)C%29)(N(C)C)=O)O[C@@H](N%44C(N=C(N)C=C%44)=O)C%28)(N(C)C)=O)O[C@@H](N%45C(N=CN=C%46N)=C%46N=C%45)C%27)(N(C)C)=O)O[C@@H](N%47C(NC(C(C)=C%47)=O)=O)C%26)(N(C)C)=O)O[C@@H](N%48C(N=C(N)C=C%48)=O)C%25)(N(C)C)=O)O[C@@H](N%49C(N=CN=C%50N)=C%50N=C%49)C%24)(N(C)C)=O)O[C@@H](N%51C(N=CN=C%52N)=C%52N=C%51)C%23)(N(C)C)=O)O[C@@H](N%53C(N=C(N)NC%54=O)=C%54N=C%53)C%22)(N(C)C)=O)O[C@@H](N%55C(N=C(N)NC%56=O)=C%56N=C%55)C%21)(N(C)C)=O)O[C@@H](N%57C(N=CN=C%58N)=C%58N=C%57)C%20)(N(C)C)=O)O[C@@H](N%59C(N=CN=C%60N)=C%60N=C%59)C%19)(N(C)C)=O)O[C@@H](N%61C(N=C(N)NC%62=O)=C%62N=C%61)C%18)(N(C)C)=O)O[C@@H](N%63C(N=CN=C%64N)=C%64N=C%63)C%17)(N(C)C)=O)O[C@@H](N%65C(NC(C(C)=C%65)=O)=O)C%16)(N(C)C)=O)O[C@@H](N%66C(N=C(N)NC%67=O)=C%67N=C%66)C%15)(N(C)C)=O)O[C@@H](N%68C(N=C(N)NC%69=O)=C%69N=C%68)C%14)(N(C)C)=O)O[C@@H](N%70C(N=C(N)C=C%70)=O)C%13)(N(C)C)=O)O[C@@H](N%71C%72=C(N=C%71)C(N)=NC=N%72)C%12)(N(C)C)=O)O[C@@H](N%73C(NC(C(C)=C%73)=O)=O)C%11)(N(C)C)=O)O[C@@H](N%74C(NC(C(C)=C%74)=O)=O)C%10)(N(C)C)=O)O[C@@H](N%75C(NC(C(C)=C%75)=O)=O)C9)(N(C)C)=O)O[C@@H](N%76C(N=C(N)C=C%76)=O)C8)(N(C)C)=O)O[C@@H](N%77C(NC(C(C)=C%77)=O)=O)C7)(N(C)C)=O)(N(C)C)=O
Biological Activity: Eteplirsen (AVI 4658) is a phosphorylated diamine morpholino oligonucleotide that targets exon 51 of the human Duchenne muscular dystrophy (DMD) gene. Eteplirsen induces exon 51 skipping, causing it to be skipped during splicing, thereby restoring the translation reading frame and producing a shortened functional dystrophin. Eteplirsen can be used in research on Duchenne muscular dystrophy[1][2][3][4].
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Eteplirsen | 98.50% | Eteplirsen (AVI 4658) is a phosphorylated diamine morpholino oligonucleotide that targets exon 51 of the human Duchenne muscular dystrophy (DMD) gene. Eteplirsen induces exon 51 skipping, causing it to be skipped during splicing, thereby restoring the translation reading frame and producing a shortened functional dystrophin. Eteplirsen can be used in research on Duchenne muscular dystrophy. | ||||||||||||||||||||
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- [1]. Lim KR, et al. Eteplirsen in the treatment of Duchenne muscular dystrophy. Drug Des Devel Ther. 2017 Feb 28;11:533-545. [Content Brief]
- [2]. Sazani P, et al. Repeat-dose toxicology evaluation in cynomolgus monkeys of AVI-4658, a phosphorodiamidate morpholino oligomer (PMO) drug for the treatment of duchenne muscular dystrophy. Int J Toxicol. 2011 May;30(3):313-21. [Content Brief]
- [3]. Sazani P, et al. Safety pharmacology and genotoxicity evaluation of AVI-4658. Int J Toxicol. 2010 Mar-Apr;29(2):143-56. [Content Brief]
- [4]. Shimo T, et al. Construction of a tri-chromatic reporter cell line for the rapid and simple screening of splice-switching oligonucleotides targeting DMD exon 51 using high content screening. PLoS One. 2018 May 16;13(5):e0197373. [Content Brief]
Keywords
