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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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Detalles Bibliográficos
Autores principales:	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
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Oxford University Press 2021
Materias:	Chemical Biology and Nucleic Acid Chemistry
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8216463/ https://www.ncbi.nlm.nih.gov/pubmed/34107015 http://dx.doi.org/10.1093/nar/gkab488

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