Ribosomal Frameshifting Selectively Modulates the Assembly, Function, and Pharmacological Rescue of a Misfolded CFTR Variant
P. Carmody, F. J. Roushar, A. Tedman, W. Wang, M. Herwig, M. Kim, E. F. McDonald, K. Noguera, J. Wong-Roushar, J.-L. Poirier, N. Zelt, B. Pockrass, A. G. McKee, C. P. Kuntz, S. V. Raju, , J. P. Schlebach
This paper identifies an RNA structure in the CFTR transcript that promotes ribosomal frameshifting and premature termination, enhancing the function and pharmacological rescue of the misfolded {Delta}F508 CFTR variant.
Proceedings of the National Academy of Sciences · Oct 10, 2024Peer-reviewedBiochemistry