Research ArticleCystic Fibrosis

Characterizing responses to CFTR-modulating drugs using rectal organoids derived from subjects with cystic fibrosis

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Science Translational Medicine  22 Jun 2016:
Vol. 8, Issue 344, pp. 344ra84
DOI: 10.1126/scitranslmed.aad8278

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Mini-guts for personalized cystic fibrosis therapy

Cystic fibrosis is caused by mutations in the CFTR gene that severely reduce the function of the CFTR protein. New drugs for treating cystic fibrosis modulate CFTR protein function, but drug efficacy is dependent on which CFTR mutation a patient carries. Dekkers et al. now show that the efficacy of these drugs can be individually assessed in a laboratory test using epithelial cells cultured as mini-guts from rectal biopsies from subjects with cystic fibrosis. The authors show that the drug responses observed in mini-guts or rectal organoids can be used to predict which patients may be potential responders to the drug. This preclinical test may help to quickly identify responders to CFTR-modulating drug therapy even when patients carry very rare CFTR mutations.