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Eteplirsen (AVI 4658) is a synthetic antisense oligonucleotide employed in Duchenne muscular dystrophy research.
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Description | Eteplirsen (AVI 4658) is a synthetic antisense oligonucleotide employed in Duchenne muscular dystrophy research. |
In vitro | Eteplirsen functions by enhancing dystrophin synthesis, thereby treating Duchenne muscular dystrophy (DMD). It achieves this by selectively skipping exon 51 in mutated gene variants, which restores the translational reading frame necessary for proper protein production[1]. |
Alias | AVI 4658 |
Cas No. | 1173755-55-9 |
Storage | Powder: -20°C for 3 years | In solvent: -80°C for 1 year | Shipping with blue ice. |
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