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Twenty-four ASOs were designed to target the mouse mutant Notch3 pre-mRNA specifically and induce degradation of the LMS-causing transcript. Grey bars represent the site targeted by each ASO. The colored letters inside the grey bars represent the residues that are mutated in the LMS compared to the wild-type mouse Notch3 transcript. The wild-type mouse Notch3 pre-mRNA sequence is reported at top of the figure. Arrows indicate the three ASOs studied in detail (ASO 14, 17 and 18).
