2024
DOI: 10.1101/2024.03.13.584768
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Benchmarking organ-specific responses to therapies in tissues differentiated from Cystic Fibrosis patient derived iPSCs

Abdelkader Daoud,
Sunny Xia,
Onofrio Laselva
et al.

Abstract: Cystic Fibrosis (CF) is a life-shortening disease that is caused by mutations in theCFTRgene, a gene that is expressed in multiple organs. There are several primary tissue models of CF disease, including nasal epithelial cultures and rectal organoids, that are effective in reporting the potential efficacy of mutation-targeted therapies called CFTR modulators. However, there is the well-documented variation in tissue dependent, therapeutic response amongst CF patients, even those with the same CF-causing mutati… Show more

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