Article

Benchmarking organ-specific responses to therapies in tissues differentiated from Cystic Fibrosis patient derived iPSCs

2024-03-14

Abstract excerpt

Cystic Fibrosis (CF) is a life-shortening disease that is caused by mutations in the CFTR gene, 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 t...

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Literature Corpus work: 5f2f3b96-9137-5782-ac57-a4abd731189c
DOI: 10.1101/2024.03.13.584768

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