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Enhanced delivery of peptide-morpholino oligonucleotides with a small molecule to correct splicing defects in the lung

Pulmonary diseases offer many targets for oligonucleotide therapeutics. However, effective delivery of oligonucleotides to the lung is challenging. For example, splicing mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) affect a significant cohort of Cystic Fibrosis (CF) pa...

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Bibliografiske detaljer
Udgivet i:Nucleic Acids Res
Main Authors: Dang, Yan, van Heusden, Catharina, Nickerson, Veronica, Chung, Felicity, Wang, Yang, Quinney, Nancy L, Gentzsch, Martina, Randell, Scott H, Moulton, Hong M, Kole, Ryszard, Ni, Aiguo, Juliano, Rudolph L, Kreda, Silvia M
Format: Artigo
Sprog:Inglês
Udgivet: Oxford University Press 2021
Fag:
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC8216463/
https://ncbi.nlm.nih.gov/pubmed/34107015
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/nar/gkab488
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