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Pharmacol."],"abstract":"<jats:sec>\n                    <jats:title>Rationale<\/jats:title>\n                    <jats:p>Respiratory status of people with Cystic Fibrosis (pwCF) carrying N1303K is improved by Elexacaftor\/Tezacaftor\/Ivacaftor (ETI) but, contrary to other mutations, the impact on sweat test results is limited.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>\n                      To explore this discrepancy, we implemented new sweat gland and respiratory cell lines stably expressing Wild type (WT)-, F508del- and N1303K-CFTR. CFTR dependent chloride (Cl\n                      <jats:sup>\u2212<\/jats:sup>\n                      ) and bicarbonate (HCO\n                      <jats:sub>3<\/jats:sub>\n                      -) transport was measured by short circuit current in these new models and in primary Human Nasal Epithelial Cells (HNECs). CFTR expression was evaluated by Western blot.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      In the airway and the sweat gland cells expressing F508del-CFTR, ETI induced maturation of CFTR and increased Cl\n                      <jats:sup>\u2212<\/jats:sup>\n                      transport. In the respiratory cell lines and HNECs, N1303K-CFTR generated both immature and mature forms of CFTR. Correction by ETI increased CFTR amounts without promoting its maturation and improved Cl\n                      <jats:sup>\u2212<\/jats:sup>\n                      secretion. N1303K-CFTR channel activity was markedly increased by co-potentiation of IVA with Apigenin. In the sweat gland, N1303K-CFTR was expressed as a globally misfolded protein, non-rescuable by ETI. API treatment to 2 patients improved FEV1 without lowering sweat Cl- content.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion<\/jats:title>\n                    <jats:p>N1303K-CFTR shows tissue specific correction and suboptimal response to ETI which can be improved by API.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.3389\/fphar.2025.1661417","type":"journal-article","created":{"date-parts":[[2025,10,29]],"date-time":"2025-10-29T07:07:32Z","timestamp":1761721652000},"update-policy":"https:\/\/doi.org\/10.3389\/crossmark-policy","source":"Crossref","is-referenced-by-count":10,"title":["Beyond Trikafta: new models to assess tissue dependent rescue of 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