Chimeras of Cell-Penetrating Peptides Demonstrate Synergistic Improvement in Antisense Efficacy

Phosphorodiamidate morpholino oligonucleotides (PMOs) make up a promising class of therapeutics for genetic disease. PMOs designed for "exon skipping" must be internalized into cells, reach the nucleus, and act on pre-mRNA to mediate their effects. One tactic for improving PMO delivery and...

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Bibliographic Details
Main Authors: Fadzen, Colin M., Holden, Rebecca Lynn, Wolfe, Justin, Choo, Zi-Ning, Schissel, Carly K., Yao, Monica, Hanson, Gunnar J., Pentelute, Bradley L.
Other Authors: Massachusetts Institute of Technology. Center for Environmental Health Sciences
Format: Article
Language:English
Published: American Chemical Society (ACS) 2020
Online Access:https://hdl.handle.net/1721.1/128007