Extract
Recent advances in adult stem cell biology have resulted in the development of organoid culture technologies using a variety of tissue sources such as intestine, lung and kidney [1]. Organoids are three-dimensional, multicellular structures that recapitulate tissue features of the parental organ and are usually grown from donor tissue fragments [1]. As organoids are functional expressions of individual genomes, these cultures are particularly useful to understand how genetic factors contribute to individual disease. As such, they are used to study hereditary diseases such as cystic fibrosis (CF), and more common diseases such as cancer where genetics can influence disease severity and drug efficacy [2, 3].
Abstract
CF can be studied using patient-specific intestinal organoids. CFTR protein function in intestinal organoids correlates with clinical disease severity and drug testing in organoids could aid in finding drugs for people with rare CFTR mutations. https://bit.ly/2GryRl0
Footnotes
Conflict of interest: P. van Mourik has nothing to disclose.
Conflict of interest: J.M. Beekman has a patent on organoid swelling for personalised diagnosis in CF with royalties paid by Hubrecht Organoid Technology. He received financial support to cover travel costs and preparation time of scientific lectures from Vertex. He also received funding from Galapagos for CFTR modulator studies.
Conflict of interest: C.K. van der Ent reports grants from Vertex Pharmaceuticals Inc, Gilead Sciences and Galapagos, grants and other funding from ProQR, and other funding from TEVA pharmaceutical industries, outside the submitted work.
- Received December 15, 2018.
- Accepted April 7, 2019.
- Copyright ©ERS 2019
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