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ASOs are designed to skip the exon where mutations occur, in the hopes of restoring the proteins function albeit partially

[DOI] [PMC free article] [PubMed] [Google Scholar] 10.Rashad A., Rasool A., Shaheryar M., Sarfraz A., Sarfraz Z., Robles-Velasco K., Cherrez-Ojeda I

[DOI] [PMC free article] [PubMed] [Google Scholar] 158.Salvio G., Cutini M., Ciarloni A., Giovannini L., Perrone M., Balercia G
