Article

Durable reconstitution of sinonasal epithelium by transplant of CFTR gene corrected airway stem cells

2025-01-26

Abstract excerpt

Modulator agents that restore cystic fibrosis transmembrane conductance regulator (CFTR) function have revolutionized outcomes in cystic fibrosis, an incurable multisystem disease. Barriers exist to modulator use, making local CFTR gene and cell therapies attractive, especially in the respiratory tract. We used CRISPR to gene-correct CFTR in upper airway basal stem cells (UABCs) and show durable local engraftment...

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Literature Corpus work: 2b2f0038-cacd-5cd9-8538-43be5a3873a8
DOI: 10.1101/2025.01.24.634776

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