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Science 293 (5534), 14951499
ASOs are designed to skip the exon where mutations occur, in the hopes of restoring the proteins function albeit partially
Ding XQ, Maudsley AA, Schweiger U, Schmitz B, Lichtinghagen R, Bleich S, et al
They're not really a function of the dose being too high or your serum B12 level being too high
