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...
Main Authors: | , , , , , , , |
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Other Authors: | |
Format: | Article |
Language: | English |
Published: |
American Chemical Society (ACS)
2020
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Online Access: | https://hdl.handle.net/1721.1/128007 |