Article
Enhanced delivery of peptide-morpholino oligonucleotides with a small molecule to correct splicing defects in the lung.
Nucleic acids research - 21 Jun 2021
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
Abstract excerpt
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) patients. These individuals could potentially benefit from treatment with splice switching oligonucleotides (SSOs)...
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