Referring to Slide 4 of the FNA Presentation…
Of the conventional CPPs against which PYC’s CPPs were measured, Pip-6a appears to have performed slightly better than β-peptide. But both were completely outclassed in efficiency by PYC’s 1st gen CPPs and even more so by PYC’s 2nd gen CPPs.
Pip6a-PMO was developed at Oxford's Department of Physiology, Anatomy and Genetics. It was initially designed for DMD but has demonstrated successful results in an SMA mouse model as well. In fact, it was concluded that
The potent systemic efficacy of Pip6a-PMO, targeting both peripheral as well as CNS tissues, demonstrates the high clinical potential of peptide-PMO therapy for SMA.
I'm looking forward to those 2nd gen in vivo results.
https://www.sciencedaily.com/releases/2016/09/160920154551.htm
http://www.pnas.org/content/113/39/10962
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