Article
Open reading frame correction using splice-switching antisense oligonucleotides for the treatment of cystic fibrosis.
Proceedings of the National Academy of Sciences of the United States of America - 18 Jan 2022
Michaels Wren E, Pena-Rasgado Cecilia, Kotaria Rusudan, Bridges Robert J, Hastings Michelle L
Abstract excerpt
CFTR gene mutations that result in the introduction of premature termination codons (PTCs) are common in cystic fibrosis (CF). This mutation type causes a severe form of the disease, likely because of low CFTR messenger RNA (mRNA) expression as a result of nonsense-mediated mRNA decay, as well as the production of a nonfunctional, truncated CFTR protein. Current therapeutics for CF, which target residual protein...
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