Pseudoexon exclusion by antisense therapy in methylmalonic aciduria (MMAuria).


Development of pseudoexon exclusion therapies by antisense modification of pre-mRNA splicing represents a type of personalized genetic medicine. Here we present the cellular antisense therapy and the cell-based splicing assays to investigate the effect of two novel deep intronic changes c.1957-898A>G and c.1957-920C>A identified in the methylmalonyl… (More)
DOI: 10.1002/humu.21118


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