Forskolin-induced swelling of intestinal organoids correlates with disease severity in adults with cystic fibrosis and homozygous F508del mutations.
de Winter-de Groot KM, Berkers G, Marck-van der Wilt REP, van der Meer R, Vonk A, Dekkers JF, Geerdink M, Michel S, Kruisselbrink E, Vries R, Clevers H, Vleggaar FP, Elias SG, Heijerman HGM, van der Ent CK, Beekman JM.
de Winter-de Groot KM, et al. Among authors: heijerman hgm.
J Cyst Fibros. 2020 Jul;19(4):614-619. doi: 10.1016/j.jcf.2019.10.022. Epub 2019 Nov 15.
J Cyst Fibros. 2020.
PMID: 31735562
Free article.
These findings suggest that differences at low CFTR residual function may contribute to clinical heterogeneity in F508del homozygous patients and small changes in CFTR residual function might impact long-term disease expression....
These findings suggest that differences at low CFTR residual function may contribute to clinical heterogeneity in F508del homozygous patient …