Steric Inhibition of 5′ UTR Regulatory Elements Results in Upregulation of Human CFTR

Cystic fibrosis (CF) is an autosomal recessive monogenic disease caused by mutations in the CFTR gene. Therapeutic approaches that are focused on correcting CFTR protein face the challenge of the heterogeneity in CFTR mutations and resulting defects. Thus, while several small molecules directed at CFTR show benefit in the clinic for subsets of CF patients, these drugs cannot treat all CF patients. Additionally, the clinical benefit from treatment with these modulators could be enhanced with novel therapies.

Citation

Shruti Sasaki, Rachel Sun, Huynh-Hoa Bui, Jeff R. Crosby, Brett P. Monia, Shuling Guo. Steric Inhibition of 5′ UTR Regulatory Elements Results in Upregulation of Human CFTR Molecular Therapy. 2019 Jul 12;27(10):1749-1757

PMID: 31351782

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