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Make or break for first splice-modulating agents

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If Duchenne muscular dystrophy drugs that modulate gene splicing can overcome key hurdles, they could boost enthusiasm for related antisense oligonucleotides in other indications.

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Figure 1: The potential of splice-modulating antisense oligonucleotides.
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Mullard, A. Make or break for first splice-modulating agents. Nat Rev Drug Discov 12, 813–815 (2013). https://doi.org/10.1038/nrd4151

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