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We fused a signal peptide (SP) and a furin-cleavable sequence (FCS) to the upstream region of the Exe4 gene to enable Exe4 secretion from the edited liver

Back Nair, P (2024) 'QnAs with Svetlana Mojsov, Joel Habener, and Lotte Bjerre Knudsen: Winners of the 2024 Lasker~DeBakey Clinical Medical Research Award', Proceedings of the National Academy of Sciences , 121/39, e2416868121

Astrup A, Rossner S, Van GL, Rissanen A, Niskanen L, Al HM et al

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Almost every dose produces a decimal that does not align cleanly with syringe markings

Notably, the placebo group showed no statistically significant cholesterol level changes