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See Latest ArticlesExon skipping-mediated dystrophin reading frame restoration for small mutations
Human Mutation, 09/29/09
Spitali P et al. – The obtained results confirm cell–free splicing assay as an alternative system to test exon skipping propensity when patients' cells are unavailable. This behavior may be related to the effect of the mutations on exon skipping propensity, and highlights the complexity of identifying optimal AONs for skipping exons with small mutations.
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