The thing that strikes me about this is the potential for this to be a disease curing conveyer belt. Find a target organ identify a disease with a single strand mutation and an under expressed protein. Optimise the CPP PMO combination. Run the trials. 62 percent chance of success. I don’t mean to downplay or be flippant about the science I just mean they could already have a large list of targets organised in order of priority. Once revenue is coming in the potential could be enormous. Am I being overly optimistic?
Ann: Polycystic Kidney Disease Program Presentation, page-8
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