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Fig. 1 | Molecular and Cellular Pediatrics

Fig. 1

From: Pluripotent stem cells for disease modeling and drug screening: new perspectives for treatment of cystic fibrosis?

Fig. 1

Generation and use of CF-disease-specific iPS cells for drug screening and disease modeling. Adult somatic cells (e.g., from blood) from any patient can be reprogrammed into induced pluripotent stem (iPS) cells. These cells can be genetically modified, expanded, and differentiated into the cell types of interest and used (i) to understand the molecular mechanisms underlying disease phenotypes, for example, the molecular causes for different clinical phenotypes in CF patients with similar mutations, and (ii) in drug screening and discovery, to determine the effects of candidate drugs and new compounds, and to identify target pathways

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