Article
Amphiphilic shuttle peptide delivers base editor ribonucleoprotein to correct the CFTR R553X mutation in well-differentiated airway epithelial cells.
Nucleic acids research - 28 Oct 2024
Kulhankova Katarina, Cheng Anna X, Traore Soumba, Auger Maud, Pelletier Mia, Hervault Maxime, Wells Kevin D, Green Jonathan A, Byrne Addison, Nelson Benjamin, Sponchiado Mariana, Boosani Chandra, Heffner Caleb S, Snow Kathy J, Murray Stephen A, Villacreses Raul A, Rector Michael V, Gansemer Nicholas D, Stoltz David A, Allamargot Chantal, Couture Frédéric, Hemez Colin, Hallée Stéphanie, Barbeau Xavier, Harvey Mario, Lauvaux Coraline, Gaillet Bruno, Newby Gregory A, Liu David R, McCray Paul B, Guay David
Abstract excerpt
Base editing could correct nonsense mutations that cause cystic fibrosis (CF), but clinical development is limited by the lack of delivery methods that efficiently breach the barriers presented by airway epithelia. Here, we present a novel amphiphilic shuttle peptide based on the previously reported S10 peptide that substantially improved base editor ribonucleoprotein (RNP) delivery. Studies of the S10 secondary...
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