Cystic fibrosis transmembrane conductance regulator (CFTR)-targeted therapeutics for cystic fibrosis

Treatment of the common life-limiting genetic disease cystic fibrosis (CF) has been transformed by small molecule therapeutics that target the root cause of the disease, pathogenic variants in the ATP-binding cassette transporter cystic fibrosis transmembrane conductance regulator (CFTR). Here, we s...

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Detalles Bibliográficos
Autores principales: Veselu, D-F, Yeh, H-I, Rodrat, M, Bradbury, JD, Manzanares Fornies, Y, Li, H, Shoemark, DK, Oliveira, ASF, Hwang, T-C, Sheppard, DN
Otros Autores: Stephens, G
Formato: Book section
Lenguaje:English
Publicado: Springer 2024