Peter van Mourik

135 Comparing in vitro and in vivo effects of lumacaftor-ivacaftor REFERENCES 1. Mall MA, Mayer-Hamblett N, Rowe SM. Cystic Fibrosis: Emergence of Highly Effective Targeted Therapeutics and Potential Clinical Implications. Am J Respir Crit Care Med 2020; 201: 1193-1208. 2. Bell SC, Mall MA, Gutierrez H, Macek M, Madge S, Davies JC, Burgel PR, Tullis E, Castanos C, Castellani C, Byrnes CA, Cathcart F, Chotirmall SH, Cosgriff R, Eichler I, Fajac I, Goss CH, Drevinek P, Farrell PM, Gravelle AM, Havermans T, Mayer-Hamblett N, Kashirskaya N, Kerem E, Mathew JL, McKone EF, Naehrlich L, Nasr SZ, Oates GR, O’Neill C, Pypops U, Raraigh KS, Rowe SM, Southern KW, Sivam S, Stephenson AL, Zampoli M, Ratjen F. The future of cystic fibrosis care: a global perspective. Lancet Respir Med 2020; 8: 65-124. 3. de Winter-de Groot KM, Janssens HM, van Uum RT, Dekkers JF, Berkers G, Vonk A, Kruisselbrink E, Oppelaar H, Vries R, Clevers H, Houwen RHJ, Escher JC, Elias SG, de Jonge HR, de Rijke YB, Tiddens HAWM, van der Ent CK, Beekman JM. Stratifying infants with cystic fibrosis for disease severity using intestinal organoid swelling as a biomarker of CFTR function. Eur Respir J 2018;52:1702529. 4. Dekkers JF, Berkers G, Kruisselbrink E, Vonk A, de Jonge HR, Janssens HM, Bronsveld I, van de Graaf EA, Nieuwenhuis EES, Houwen RHJ, Vleggaar FP, Escher JC, de Rijke YB, Majoor CJ, Heijerman HGM, de Winter-de Groot KM, Clevers H, van der Ent CK, Beekman JM. Characterizing responses to CFTR- modulating drugs using rectal organoids derived from subjects with cystic fibrosis. Sci Transl Med 2016;8:344ra84. 5. Berkers G, van Mourik P, Vonk AM, Kruisselbrink E, Dekkers JF, de Winter-de Groot KM, Arets HGM, Marck-van der Wilt REP, Dijkema JS, Vanderschuren MM, Houwen RHJ, Heijerman HGM, van de Graaf EA, Elias SG, Majoor CJ, Koppelman GH, Roukema J, Bakker M, Janssens HM, van der Meer R, Vries RGJ, Clevers HC, de Jonge HR, Beekman JM, van der Ent CK. Rectal Organoids Enable Personalized Treatment of Cystic Fibrosis. Cell Rep 2019;26:1701-1708. e3. 6. Graeber SY, Dopfer C, Naehrlich L, Gyulumyan L, Scheuermann H, Hirtz S, Wege S, Mairbäurl H, Dorda M, Hyde R, Bagheri-Hanson A, Rueckes-Nilges C, Fischer S, Mall MA, Tümmler B. Effects of Lumacaftor-Ivacaftor Therapy on Cystic Fibrosis Transmembrane Conductance Regulator Function in Phe508del Homozygous Patients with Cystic Fibrosis. Am J Respir Crit Care Med 2018;197:1433–1442. 7. Graeber SY, Hug MJ, Sommerburg O, Hirtz S, Hentschel J, Heinzmann A, Dopfer C, Schulz A, Mainz JG, Tümmler B, Mall MA. Intestinal Current Measurements Detect Activation of Mutant CFTR in Patients with Cystic Fibrosis with the G551D Mutation Treated with Ivacaftor. Am J Respir Crit Care Med 2015;192:1252–5. 6