Currently, there are no effective therapies for cystic fibrosis for patients carrying CFTR mutations. Therefore, there is an urgent need to develop relevant models with the translational potential to develop targeted therapies. Here, patient-derived human induced pluripotent stem cells are used to obtain lung progenitor cells to build a high-throughput fluorescence assay of CFTR channel activity as a platform for drug development. The results confirmed the suitability of this platform for testing studies in stem cell-derived progenitors and matched patients nasal cultures. Furthermore, positive results in the newly developed platform could be translated in primary nasal cultures from the same patients. Overall, the researchers propose a new platform to perform patient-oriented drug screening in a relevant in vitro model for cystic fibrosis.
A new platform for high-throughput therapy testing on iPSC-derived lung progenitor cells from cystic fibrosis patients
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