Novel Correctors and Potentiators Enhance Translational Readthrough in CFTR Nonsense Mutations

Premature-termination codons (PTCs) in CFTR (cystic fibrosis [CF] transmembrane conductance regulator) result in nonfunctional CFTR protein and are the proximate cause of ∼11% of CF-causing alleles, for which no treatments exist. The CFTR corrector lumacaftor and the potentiator ivacaftor improve CF...

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Detaylı Bibliyografya
Yayımlandı:Am J Respir Cell Mol Biol
Asıl Yazarlar: Mutyam, Venkateshwar, Sharma, Jyoti, Li, Yao, Peng, Ning, Chen, Jianguo, Tang, Li Ping, Falk Libby, Emily, Singh, Ashvani K., Conrath, Katja, Rowe, Steven M.
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: American Thoracic Society 2021
Konular:
Original Research
Online Erişim:https://ncbi.nlm.nih.gov/pmc/articles/PMC8086047/
https://ncbi.nlm.nih.gov/pubmed/33616476
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1165/rcmb.2019-0291OC
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