Article

Correcting mutant CFTR with a stabilizing nanobody reveals a novel active conformation of the channel

2026-07-29

Abstract excerpt

<title>Abstract</title> <p>Defects in protein trafficking underlie many genetic diseases, including cystic fibrosis (CF), where the predominant F508del mutation destabilizes the cystic fibrosis transmembrane conductance regulator (CFTR) channel, leading to its degradation. To provide a protein-specific chaperone, we used lipid nanoparticles to deliver mRNA encoding T2a, a nanobody that thermally stabilizes CFTR v...

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Literature Corpus work: dfc61648-5811-5279-a2f6-6425acab59af
DOI: 10.21203/rs.3.rs-10314869/v1

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