Journal
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
Volume 22, Issue 9, Pages -Publisher
MDPI
DOI: 10.3390/ijms22094448
Keywords
cystic fibrosis; CFTR; nasal cell; theratyping; personalized medicine
Funding
- Cincinnati Children's Research Foundation
- Cystic Fibrosis Foundation [BREWIN20Y2-OUT, BREWIN20A0-KB, KEEGAN19B0]
- National Institutes of Health [K08 HL144825-01A1]
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The emergence of highly effective CFTR modulator therapy has significantly improved healthcare for most cystic fibrosis (CF) patients. However, accessibility remains a challenge for some due to rare CFTR variants or lack of biologic activity of available therapies. The use of primary human cell-based models, particularly nasal cells, is proposed as a solution to address this gap and is crucial for personalized patient care in CF research.
The emergence of highly effective CFTR modulator therapy has led to significant improvements in health care for most patients with cystic fibrosis (CF). For some, however, these therapies remain inaccessible due to the rarity of their individual CFTR variants, or due to a lack of biologic activity of the available therapies for certain variants. One proposed method of addressing this gap is the use of primary human cell-based models, which allow preclinical therapeutic testing and physiologic assessment of relevant tissue at the individual level. Nasal cells represent one such tissue source and have emerged as a powerful model for individual disease study. The ex vivo culture of nasal cells has evolved over time, and modern nasal cell models are beginning to be utilized to predict patient outcomes. This review will discuss both historical and current state-of-the art use of nasal cells for study in CF, with a particular focus on the use of such models to inform personalized patient care.
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