Integrative chemogenomic analysis identifies small molecules that partially rescue ΔF508‐CFTR for cystic fibrosis

• Main Authors: Rachel A. Hodos (Author), Matthew D. Strub (Author), Shyam Ramachandran (Author), Ella A. Meleshkevitch (Author), Dmitri Y. Boudko (Author), Robert J. Bridges (Author), Joel T. Dudley (Author), Paul B. McCray Jr (Author)
• Format: Book
• Published: Wiley, 2021-05-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Abstract Rare diseases affect 10% of the first‐world population, yet over 95% lack even a single pharmaceutical treatment. In the present age of information, we need ways to leverage our vast data and knowledge to streamline therapeutic development and lessen this gap. Here, we develop and implement...