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...
Topics
- Alleles
- Base Sequence
- Bronchi
- Cell Line
- Chloride Channels
- Cystic Fibrosis
- Cystic Fibrosis Transmembrane Conductance Regulator
- Epithelial Cells
- Exons
- Homozygote
- Humans
- Oligonucleotides, Antisense
- Open Reading Frames