Article
Design and application of bispecific splice-switching oligonucleotides.
Nucleic acid therapeutics - 1 Feb 2014
Bestas Burcu, McClorey Graham, Tedebark Ulf, Moreno Pedro M D, Roberts Thomas C, Hammond Suzan M, Smith C I Edvard, Wood Matthew J A, Andaloussi Samir El
Abstract excerpt
Targeting of pre-mRNA by short splice-switching oligonucleotides (SSOs) is increasingly being used as a therapeutic modality, one rationale being to disrupt splicing so as to remove exons containing premature termination codons, or to restore the translation reading frame around out-of-frame deletion mutations. The aim of this study was to investigate the effect of chemically linking individual SSOs so as to...
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