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A new treatment approach for cystic fibrosis
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A new treatment approach for cystic fibrosis

by  Cold Spring Harbor Laboratory Identification of NMD-inhibiting ASOs and assessment of their specificity. a Schematic of NMD reporters. The numbers show the CFTR exons in the NMD reporters. The red asterisk (*) indicates the location of the W1282X mutation. b Schematic of ASO screening. 19 MOE-PS-modified 15-mer ASOs (yellow and magenta bars) were designed to cover the presumptive EJC binding sites...