
Dr. Dietrich Stephan: New Drugs that Repair Broken Genes
Talking Biotech with Dr. Kevin Folta
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Is the Mutin Transcript Restored?
Half is not enough of the d m pk protein itself. The other is transcribed and exported into the nucleus and forms a healthy protein, but there's not enough of it. So when we engage that target that stuck in the nucleus, that m arna, that hairpin, linearizes it. We believe that that then allows the transcript to escape into the citoplasm and be translated. And so that translationl cinery can restore the haplo insufficiency,. But why isn't this therapeutic molecule imunigenic? Ye, it's a great question. This class of compound has been reported in the academic literature to be quoted monologically inert. It's because it
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