Author
Cystic Fibrosis FoundationORCID 0000-0003-0280-9789Unclaimed ORCID profile · follow to get notified of new preprints
3 papers
Recent·Most cited
Norm Allaire, M. Armstrong, Jae Seok Yoon…·Calvin U. Cotton
The paper shows that upregulating an NMD-insensitive CFTR mRNA isoform with ASOs and ETI can restore partial Cl- transport function in cells with 3' PTC mutations.
Wooree Ko, Joseph J. Porter, Sacha Spelier…·John D. Lueck
The paper demonstrates that ACE-tRNAs can suppress nonsense mutations in CF by rescuing CFTR transcript abundance and channel function in multiple models.
Hermann J. Bihler, Andrey Sivachenko, Linda Millen…·Martin Mense
This study evaluates the responsiveness of 655 CFTR variants to the modulator therapy ELX/TEZ/IVA, identifying variants that show improved chloride transport function.