Open access
Aug 2026
A functional comparison of readthrough agent ELX-02 across a wide range of nonsense CFTR variants
M. Destoop, A. Ramalho, Iris AL. Silva et al.
· bioRxiv · 0 citations
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It is shown that spikes from both serotypes of HKU1 preferentially bind 9-O-acetylated α2,8-linked disialosides, and molecular dynamics simulations indicated that membrane-embedded GT3, but not GD3, presents its glycan chain in a geometry compatible with S1A-mediated engagement.
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