phase 2 trial

GlaxoSmithKline's Duchenne MD exon-skipping drug moves into a phase 3 trial and is renamed drisapersen; "block skipping" of exons 45-55 in mice looks promising

posted on August 15, 2012 - 2:41pm
Exon skipping is an experimental therapeutic strategy in which regions — exons — of a gene are targeted and blocked ("skipped") by laboratory-designed molecules. The goal is that the remaining genetic instructions will lead to production of a shorter but still-functional protein.

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