GenomicsbioRxivPreprint
Upregulation of a nonsense mediated decay (NMD) insensitive CFTR mRNA isoform has therapeutic potential for the treatment of 3 Prime CFTR PTC variants
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.
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