Tips to Design Effective Splice-Switching Antisense Oligonucleotides for Exon Skipping and Exon Inclusion.
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DOI: 10.1007/978-1-4939-8651-4_5
Abstract: Antisense-mediated exon skipping and exon inclusion have proven to be powerful tools for treating neuromuscular diseases. The approval of Exondys 51 (eteplirsen) and Spinraza (nusinersen) for the treatment of patients with Duchenne muscular dystrophy (DMD)… read more here.
Keywords: exon inclusion; exon skipping; skipping exon; muscular dystrophy ... See more keywords