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Integrity and Stability of PTC Bearing CFTR mRNA and Relevance to Future Modulator Therapies in Cystic Fibrosis

Major advances have recently been made in the development and application of CFTR (cystic fibrosis transmembrane conductance regulator) mutation class-specific modulator therapies, but to date, there are no approved modulators for Class I mutations, i.e., those introducing a premature termination co...

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Detalles Bibliográficos
Autores principales: Clarke, Luka A., Luz, Vanessa C. C., Targowski, Szymon, Ramalho, Sofia S., Farinha, Carlos M., Amaral, Margarida D.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8621375/
https://www.ncbi.nlm.nih.gov/pubmed/34828417
http://dx.doi.org/10.3390/genes12111810