Use of a High-Throughput Phenotypic Screening Strategy to Identify Amplifiers, a Novel Pharmacological Class of Small Molecules That Exhibit Functional Synergy with Potentiators and Correctors

Cystic fibrosis (CF) is a lethal genetic disorder caused by mutation of the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Despite recent groundbreaking approval of genotype-specific small-molecule drugs, a significant portion of CF patients still lack effective therapeutic options...

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Detalles Bibliográficos
Autores principales: Giuliano, Kenneth A., Wachi, Shinichiro, Drew, Lawrence, Dukovski, Danijela, Green, Olivia, Bastos, Cecilia, Cullen, Matthew D., Hauck, Sheila, Tait, Bradley D., Munoz, Benito, Lee, Po-Shun, Miller, John Preston
Formato: Online Artículo Texto
Lenguaje:English
Publicado: SAGE Publications 2017
Materias:
Original Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5784457/
https://www.ncbi.nlm.nih.gov/pubmed/28898585
http://dx.doi.org/10.1177/2472555217729790

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