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AVI's morpholino antisense oligomers appear to be the most efficient chemistry approach for exon skipping. The antisense oligomers target and disable essential bacterial gene function, and the delivery peptides improve the antisense uptake into bacterial cells. Morpholino antisense oligomers appear to be the most efficient chemistry approach for exon-skipping, as they exhibit low toxicity, have been administered systemically, results persist for months in the body, and several compounds have already been used in human clinical trials. |
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