I understand that the rationale for the use of morpholinos [PMOs] in RNA therapeutics is that the design facilitates precision medicine. Once you have the basic structure and established the backbone chemistry, all you need to do is change the sequence of nucleotides for a new target address. That at least is what I have read on the topic.
The morpholino platform is not unique to PYC, however, the delivery vehicle using CPPs provides for a novel delivery mechanism. CPPs are on the rise as AAV vectors are under severe challenge. The powerful nature of the platform to offer facilitated delivery of precision medicine is the excitement. Further, the announcement of a disease indication in the CNS is eagerly anticipated.
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