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U1-mediated rescue in hemophilia A. Hemophilia A model caused by multiple nucleotide changes within the F8 exon 19. Schematic representation of the genomic context (left panel), splicing transcripts (middle panel) and protein isoforms (right panel) is reported. Frameshift of the coding sequence and premature stop codons are reported by asterisks and red X letter, respectively. Percentages of transcripts, antigen and relative coagulation activity are shown on the right.
