AAO 2020 poster by Stephen Russel et al. Recognized with best poster award. Sepofarsen had a manageable safety profile and showed improvement in mean visual acuity, and full-field stimulus testing.
ARVO 2020 clinical trial results presentation by Stephen Russel et al. Sepofarsen had a manageable safety profile and showed improvement in mean visual acuity, and full-field stimulus testing.
Modification of a guide RNA can mimic the effect of hyperactive mutants and advance the approach of recruiting endogenous ADARs for site-directed RNA editing reported in Journal of the American Chemical Society (JACS) by Doherty EE et al.