المؤلفون: Rodenburg LW; Department of Pediatric Pulmonology, Wilhelmina Children's Hospital, University Medical Center Utrecht, Utrecht University, Member of ERN-LUNG, 3584 EA, Utrecht, The Netherlands. l.w.rodenburg-2@umcutrecht.nl.; Regenerative Medicine Center Utrecht, University Medical Center Utrecht, Utrecht University, 3584 CT, Utrecht, The Netherlands. l.w.rodenburg-2@umcutrecht.nl., Metzemaekers M; Department of Pulmonary Medicine, Erasmus University Medical Center, 3015 CE, Rotterdam, The Netherlands.; Department of Cell Biology, Erasmus University Medical Center, 3015 CE, Rotterdam, The Netherlands., van der Windt IS; Department of Pediatric Pulmonology, Wilhelmina Children's Hospital, University Medical Center Utrecht, Utrecht University, Member of ERN-LUNG, 3584 EA, Utrecht, The Netherlands.; Regenerative Medicine Center Utrecht, University Medical Center Utrecht, Utrecht University, 3584 CT, Utrecht, The Netherlands., Smits SMA; Department of Pediatric Pulmonology, Wilhelmina Children's Hospital, University Medical Center Utrecht, Utrecht University, Member of ERN-LUNG, 3584 EA, Utrecht, The Netherlands.; Regenerative Medicine Center Utrecht, University Medical Center Utrecht, Utrecht University, 3584 CT, Utrecht, The Netherlands., den Hertog-Oosterhoff LA; Department of Pediatric Pulmonology, Wilhelmina Children's Hospital, University Medical Center Utrecht, Utrecht University, Member of ERN-LUNG, 3584 EA, Utrecht, The Netherlands.; Regenerative Medicine Center Utrecht, University Medical Center Utrecht, Utrecht University, 3584 CT, Utrecht, The Netherlands., Kruisselbrink E; Department of Pediatric Pulmonology, Wilhelmina Children's Hospital, University Medical Center Utrecht, Utrecht University, Member of ERN-LUNG, 3584 EA, Utrecht, The Netherlands.; Regenerative Medicine Center Utrecht, University Medical Center Utrecht, Utrecht University, 3584 CT, Utrecht, The Netherlands., Brunsveld JE; Department of Pediatric Pulmonology, Wilhelmina Children's Hospital, University Medical Center Utrecht, Utrecht University, Member of ERN-LUNG, 3584 EA, Utrecht, The Netherlands.; Regenerative Medicine Center Utrecht, University Medical Center Utrecht, Utrecht University, 3584 CT, Utrecht, The Netherlands., Michel S; Department of Pediatric Pulmonology, Wilhelmina Children's Hospital, University Medical Center Utrecht, Utrecht University, Member of ERN-LUNG, 3584 EA, Utrecht, The Netherlands., de Winter-de Groot KM; Department of Pediatric Pulmonology, Wilhelmina Children's Hospital, University Medical Center Utrecht, Utrecht University, Member of ERN-LUNG, 3584 EA, Utrecht, The Netherlands., van der Ent CK; Department of Pediatric Pulmonology, Wilhelmina Children's Hospital, University Medical Center Utrecht, Utrecht University, Member of ERN-LUNG, 3584 EA, Utrecht, The Netherlands., Stadhouders R; Department of Pulmonary Medicine, Erasmus University Medical Center, 3015 CE, Rotterdam, The Netherlands.; Department of Cell Biology, Erasmus University Medical Center, 3015 CE, Rotterdam, The Netherlands., Beekman JM; Department of Pediatric Pulmonology, Wilhelmina Children's Hospital, University Medical Center Utrecht, Utrecht University, Member of ERN-LUNG, 3584 EA, Utrecht, The Netherlands.; Regenerative Medicine Center Utrecht, University Medical Center Utrecht, Utrecht University, 3584 CT, Utrecht, The Netherlands.; Centre for Living Technologies, Alliance TU/e, WUR, UU, UMC Utrecht, 3584 CB, Utrecht, The Netherlands., Amatngalim GD; Department of Pediatric Pulmonology, Wilhelmina Children's Hospital, University Medical Center Utrecht, Utrecht University, Member of ERN-LUNG, 3584 EA, Utrecht, The Netherlands.; Regenerative Medicine Center Utrecht, University Medical Center Utrecht, Utrecht University, 3584 CT, Utrecht, The Netherlands.

المصدر: Scientific reports [Sci Rep] 2023 Oct 30; Vol. 13 (1), pp. 18573. Date of Electronic Publication: 2023 Oct 30.

نوع المنشور: Journal Article; Research Support, Non-U.S. Gov't

بيانات الدورية: Publisher: Nature Publishing Group Country of Publication: England NLM ID: 101563288 Publication Model: Electronic Cited Medium: Internet ISSN: 2045-2322 (Electronic) Linking ISSN: 20452322 NLM ISO Abbreviation: Sci Rep Subsets: MEDLINE

مواضيع طبية MeSH: Cystic Fibrosis*/genetics , Cystic Fibrosis*/metabolism, Humans ; Cells, Cultured ; Respiratory Mucosa/metabolism ; Nose ; Epithelial Cells/metabolism ; Cystic Fibrosis Transmembrane Conductance Regulator/metabolism

المؤلفون: de Poel E; Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, EA 3584, the Netherlands; Regenerative Medicine Utrecht, University Medical Center, Utrecht University, Utrecht, CT 3584, the Netherlands., Spelier S; Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, EA 3584, the Netherlands; Regenerative Medicine Utrecht, University Medical Center, Utrecht University, Utrecht, CT 3584, the Netherlands., Hagemeijer MC; Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, EA 3584, the Netherlands; Regenerative Medicine Utrecht, University Medical Center, Utrecht University, Utrecht, CT 3584, the Netherlands; Center for Lysosomal and Metabolic Diseases, Department of Clinical Genetics, Erasmus University Medical Center, Rotterdam, GD 3015, the Netherlands., van Mourik P; Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, EA 3584, the Netherlands., Suen SWF; Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, EA 3584, the Netherlands; Regenerative Medicine Utrecht, University Medical Center, Utrecht University, Utrecht, CT 3584, the Netherlands., Vonk AM; Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, EA 3584, the Netherlands; Regenerative Medicine Utrecht, University Medical Center, Utrecht University, Utrecht, CT 3584, the Netherlands., Brunsveld JE; Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, EA 3584, the Netherlands; Regenerative Medicine Utrecht, University Medical Center, Utrecht University, Utrecht, CT 3584, the Netherlands., Ithakisiou GN; Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, EA 3584, the Netherlands; Regenerative Medicine Utrecht, University Medical Center, Utrecht University, Utrecht, CT 3584, the Netherlands., Kruisselbrink E; Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, EA 3584, the Netherlands; Regenerative Medicine Utrecht, University Medical Center, Utrecht University, Utrecht, CT 3584, the Netherlands., Oppelaar H; Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, EA 3584, the Netherlands; Regenerative Medicine Utrecht, University Medical Center, Utrecht University, Utrecht, CT 3584, the Netherlands., Berkers G; Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, EA 3584, the Netherlands., de Winter de Groot KM; Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, EA 3584, the Netherlands., Heida-Michel S; Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, EA 3584, the Netherlands., Jans SR; Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, EA 3584, the Netherlands., van Panhuis H; Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, EA 3584, the Netherlands., Bakker M; Department of Pulmonology, Erasmus MC, University Medical Center, Rotterdam, GD 3015, the Netherlands., van der Meer R; Haga Teaching Hospital, The Hague, CH 2545, the Netherlands., Roukema J; Radboud University Medical Center, Radboud Institute for Health Sciences, Nijmegen, XZ 6525, the Netherlands., Dompeling E; Maastricht University Medical Center, Maastricht, HX 6229, the Netherlands., Weersink EJM; Amsterdam University Medical Center, location AMC, Amsterdam, AZ 1105, the Netherlands., Koppelman GH; University of Groningen, University Medical Center Groningen, Beatrix Children's Hospital, Department of Pediatric Pulmonology and Pediatric Allergology, Groningen, the Netherlands; University of Groningen, University Medical Center Groningen, Groningen Research Institute for Asthma and COPD (GRIAC), Groningen, the Netherlands., Blaazer AR; Division of Medicinal Chemistry, Vrije Universiteit Amsterdam, Amsterdam, HZ 1081, the Netherlands., Muijlwijk-Koezen JE; Division of Medicinal Chemistry, Vrije Universiteit Amsterdam, Amsterdam, HZ 1081, the Netherlands., van der Ent CK; Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, EA 3584, the Netherlands., Beekman JM; Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, EA 3584, the Netherlands; Regenerative Medicine Utrecht, University Medical Center, Utrecht University, Utrecht, CT 3584, the Netherlands; Centre for Living Technologies, Alliance TU/e, WUR, UU, UMC Utrecht, Princetonlaan 6, Utrecht, CB 3584, the Netherlands. Electronic address: J.Beekman@umcutrecht.nl.

المصدر: Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society [J Cyst Fibros] 2023 May; Vol. 22 (3), pp. 548-559. Date of Electronic Publication: 2023 May 03.

نوع المنشور: Journal Article; Research Support, Non-U.S. Gov't

بيانات الدورية: Publisher: Elsevier Country of Publication: Netherlands NLM ID: 101128966 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1873-5010 (Electronic) Linking ISSN: 15691993 NLM ISO Abbreviation: J Cyst Fibros Subsets: MEDLINE

مواضيع طبية MeSH: Cystic Fibrosis*/drug therapy , Cystic Fibrosis*/genetics , Phosphodiesterase 4 Inhibitors*/therapeutic use, Humans ; Cystic Fibrosis Transmembrane Conductance Regulator/genetics ; Cystic Fibrosis Transmembrane Conductance Regulator/therapeutic use ; Drug Repositioning ; Drug Evaluation, Preclinical ; Mutation ; Colforsin ; Genotype ; Organoids

المؤلفون: Spelier S; Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, The Netherlands.; Regenerative Medicine Utrecht, University Medical Center, Utrecht University, Utrecht, The Netherlands.; These authors contributed equally to this work., de Poel E; Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, The Netherlands.; Regenerative Medicine Utrecht, University Medical Center, Utrecht University, Utrecht, The Netherlands.; These authors contributed equally to this work., Ithakisiou GN; Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, The Netherlands.; Regenerative Medicine Utrecht, University Medical Center, Utrecht University, Utrecht, The Netherlands., Suen SWF; Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, The Netherlands.; Regenerative Medicine Utrecht, University Medical Center, Utrecht University, Utrecht, The Netherlands., Hagemeijer MC; Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, The Netherlands.; Regenerative Medicine Utrecht, University Medical Center, Utrecht University, Utrecht, The Netherlands.; Center for Lysosomal and Metabolic Diseases, Department of Clinical Genetics Erasmus University Medical Center, Rotterdam, The Netherlands., Muilwijk D; Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, The Netherlands., Vonk AM; Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, The Netherlands.; Regenerative Medicine Utrecht, University Medical Center, Utrecht University, Utrecht, The Netherlands., Brunsveld JE; Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, The Netherlands.; Regenerative Medicine Utrecht, University Medical Center, Utrecht University, Utrecht, The Netherlands., Kruisselbrink E; Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, The Netherlands.; Regenerative Medicine Utrecht, University Medical Center, Utrecht University, Utrecht, The Netherlands., van der Ent CK; Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, The Netherlands., Beekman JM; Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, The Netherlands.; Regenerative Medicine Utrecht, University Medical Center, Utrecht University, Utrecht, The Netherlands.

المصدر: ERJ open research [ERJ Open Res] 2023 Jan 30; Vol. 9 (1). Date of Electronic Publication: 2023 Jan 30 (Print Publication: 2023).

نوع المنشور: Journal Article

بيانات الدورية: Publisher: European Respiratory Society Country of Publication: England NLM ID: 101671641 Publication Model: eCollection Cited Medium: Print ISSN: 2312-0541 (Print) Linking ISSN: 23120541 NLM ISO Abbreviation: ERJ Open Res Subsets: PubMed not MEDLINE

المؤلفون: Muilwijk D; Dept of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, The Netherlands.; These authors contributed equally to this work., de Poel E; Dept of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, The Netherlands.; Regenerative Medicine Utrecht, University Medical Center, Utrecht University, Utrecht, The Netherlands.; These authors contributed equally to this work., van Mourik P; Dept of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, The Netherlands., Suen SWF; Dept of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, The Netherlands.; Regenerative Medicine Utrecht, University Medical Center, Utrecht University, Utrecht, The Netherlands., Vonk AM; Dept of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, The Netherlands.; Regenerative Medicine Utrecht, University Medical Center, Utrecht University, Utrecht, The Netherlands., Brunsveld JE; Dept of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, The Netherlands.; Regenerative Medicine Utrecht, University Medical Center, Utrecht University, Utrecht, The Netherlands., Kruisselbrink E; Dept of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, The Netherlands.; Regenerative Medicine Utrecht, University Medical Center, Utrecht University, Utrecht, The Netherlands., Oppelaar H; Dept of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, The Netherlands.; Regenerative Medicine Utrecht, University Medical Center, Utrecht University, Utrecht, The Netherlands., Hagemeijer MC; Dept of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, The Netherlands.; Regenerative Medicine Utrecht, University Medical Center, Utrecht University, Utrecht, The Netherlands.; Center for Lysosomal and Metabolic Diseases, Dept of Clinical Genetics, Erasmus University Medical Center, Rotterdam, The Netherlands., Berkers G; Dept of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, The Netherlands., de Winter-de Groot KM; Dept of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, The Netherlands., Heida-Michel S; Dept of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, The Netherlands., Jans SR; Dept of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, The Netherlands., van Panhuis H; Dept of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, The Netherlands., van der Eerden MM; Dept of Pulmonology, Erasmus MC, University Medical Center, Rotterdam, The Netherlands., van der Meer R; Haga Teaching Hospital, The Hague, The Netherlands., Roukema J; Radboud University Medical Center, Radboud Institute for Health Sciences, Nijmegen, The Netherlands., Dompeling E; Maastricht University Medical Center, Maastricht, The Netherlands., Weersink EJM; Amsterdam University Medical Center, location AMC, Amsterdam, The Netherlands., Koppelman GH; University of Groningen, University Medical Center Groningen, Beatrix Children's Hospital, Department of Pediatric Pulmonology and Pediatric Allergology, Groningen, The Netherlands.; University of Groningen, University Medical Center Groningen, Groningen Research Institute for Asthma and COPD (GRIAC), Groningen, The Netherlands., Vries R; Hubrecht Organoid Technology (HUB), Utrecht, The Netherlands., Zomer-van Ommen DD; Dutch Cystic Fibrosis Foundation (NCFS), Baarn, The Netherlands., Eijkemans MJC; Dept of Data Science and Biostatistics, Julius Center for Health Sciences and Primary Care, University Medical Center, Utrecht University, Utrecht, The Netherlands., van der Ent CK; Dept of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, The Netherlands.; C.K. van der Ent and J.M. Beekman contributed equally to this article as lead authors and supervised the work., Beekman JM; Dept of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, The Netherlands J.Beekman@umcutrecht.nl.; Regenerative Medicine Utrecht, University Medical Center, Utrecht University, Utrecht, The Netherlands.; Center for Living Technologies, University Medical Center, Utrecht, The Netherlands.; C.K. van der Ent and J.M. Beekman contributed equally to this article as lead authors and supervised the work.

المصدر: The European respiratory journal [Eur Respir J] 2022 Aug 18; Vol. 60 (2). Date of Electronic Publication: 2022 Aug 18 (Print Publication: 2022).

نوع المنشور: Journal Article; Research Support, Non-U.S. Gov't

بيانات الدورية: Publisher: European Respiratory Society Country of Publication: England NLM ID: 8803460 Publication Model: Electronic-Print Cited Medium: Internet ISSN: 1399-3003 (Electronic) Linking ISSN: 09031936 NLM ISO Abbreviation: Eur Respir J Subsets: MEDLINE

مواضيع طبية MeSH: Cystic Fibrosis*/complications , Cystic Fibrosis*/genetics , Exocrine Pancreatic Insufficiency*/complications, Biomarkers ; Colforsin/pharmacology ; Cystic Fibrosis Transmembrane Conductance Regulator/genetics ; Disease Progression ; Humans ; Mutation ; Organoids

المؤلفون: Amatngalim GD; Department of Pediatric Pulmonology, Wilhelmina Children's Hospital, University Medical Center Utrecht, Utrecht University, Member of ERN-LUNG, Utrecht, The Netherlands G.D.Amatngalim@umcutrecht.nl.; Regenerative Medicine Center Utrecht, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands., Rodenburg LW; Department of Pediatric Pulmonology, Wilhelmina Children's Hospital, University Medical Center Utrecht, Utrecht University, Member of ERN-LUNG, Utrecht, The Netherlands.; Regenerative Medicine Center Utrecht, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands., Aalbers BL; Department of Pulmonology, University Medical Center Utrecht, Utrecht, The Netherlands., Raeven HH; Department of Pediatric Pulmonology, Wilhelmina Children's Hospital, University Medical Center Utrecht, Utrecht University, Member of ERN-LUNG, Utrecht, The Netherlands.; Regenerative Medicine Center Utrecht, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands., Aarts EM; Department of Pediatric Pulmonology, Wilhelmina Children's Hospital, University Medical Center Utrecht, Utrecht University, Member of ERN-LUNG, Utrecht, The Netherlands.; Regenerative Medicine Center Utrecht, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands., Sarhane D; Department of Pediatric Pulmonology, Wilhelmina Children's Hospital, University Medical Center Utrecht, Utrecht University, Member of ERN-LUNG, Utrecht, The Netherlands.; Regenerative Medicine Center Utrecht, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands., Spelier S; Department of Pediatric Pulmonology, Wilhelmina Children's Hospital, University Medical Center Utrecht, Utrecht University, Member of ERN-LUNG, Utrecht, The Netherlands.; Regenerative Medicine Center Utrecht, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands., Lefferts JW; Department of Pediatric Pulmonology, Wilhelmina Children's Hospital, University Medical Center Utrecht, Utrecht University, Member of ERN-LUNG, Utrecht, The Netherlands.; Regenerative Medicine Center Utrecht, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands., Silva IA; BioISI-Biosystems and Integrative Sciences Institute, Faculty of Sciences, University of Lisboa, Lisboa, Portugal., Nijenhuis W; Department of Biology, Cell Biology, Neurobiology and Biophysics, Faculty of Science, Utrecht University, Utrecht, The Netherlands.; Centre for Living Technologies, Eindhoven-Wageningen-Utrecht Alliance, Utrecht, The Netherlands., Vrendenbarg S; Department of Pediatric Pulmonology, Wilhelmina Children's Hospital, University Medical Center Utrecht, Utrecht University, Member of ERN-LUNG, Utrecht, The Netherlands.; Regenerative Medicine Center Utrecht, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands., Kruisselbrink E; Department of Pediatric Pulmonology, Wilhelmina Children's Hospital, University Medical Center Utrecht, Utrecht University, Member of ERN-LUNG, Utrecht, The Netherlands.; Regenerative Medicine Center Utrecht, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands., Brunsveld JE; Department of Pediatric Pulmonology, Wilhelmina Children's Hospital, University Medical Center Utrecht, Utrecht University, Member of ERN-LUNG, Utrecht, The Netherlands.; Regenerative Medicine Center Utrecht, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands., van Drunen CM; Department of Otorhinolaryngology, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam, The Netherlands., Michel S; Department of Pediatric Pulmonology, Wilhelmina Children's Hospital, University Medical Center Utrecht, Utrecht University, Member of ERN-LUNG, Utrecht, The Netherlands., de Winter-de Groot KM; Department of Pediatric Pulmonology, Wilhelmina Children's Hospital, University Medical Center Utrecht, Utrecht University, Member of ERN-LUNG, Utrecht, The Netherlands., Heijerman HG; Department of Pulmonology, University Medical Center Utrecht, Utrecht, The Netherlands., Kapitein LC; Department of Biology, Cell Biology, Neurobiology and Biophysics, Faculty of Science, Utrecht University, Utrecht, The Netherlands.; Centre for Living Technologies, Eindhoven-Wageningen-Utrecht Alliance, Utrecht, The Netherlands., Amaral MD; BioISI-Biosystems and Integrative Sciences Institute, Faculty of Sciences, University of Lisboa, Lisboa, Portugal., van der Ent CK; Department of Pediatric Pulmonology, Wilhelmina Children's Hospital, University Medical Center Utrecht, Utrecht University, Member of ERN-LUNG, Utrecht, The Netherlands., Beekman JM; Department of Pediatric Pulmonology, Wilhelmina Children's Hospital, University Medical Center Utrecht, Utrecht University, Member of ERN-LUNG, Utrecht, The Netherlands.; Regenerative Medicine Center Utrecht, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands.; Centre for Living Technologies, Eindhoven-Wageningen-Utrecht Alliance, Utrecht, The Netherlands.

المصدر: Life science alliance [Life Sci Alliance] 2022 Aug 03; Vol. 5 (12). Date of Electronic Publication: 2022 Aug 03.

نوع المنشور: Journal Article; Research Support, Non-U.S. Gov't

بيانات الدورية: Publisher: Life Science Alliance, LLC Country of Publication: United States NLM ID: 101728869 Publication Model: Electronic Cited Medium: Internet ISSN: 2575-1077 (Electronic) Linking ISSN: 25751077 NLM ISO Abbreviation: Life Sci Alliance Subsets: MEDLINE

مواضيع طبية MeSH: Cystic Fibrosis*/genetics, Cells, Cultured ; Cystic Fibrosis Transmembrane Conductance Regulator/genetics ; Epithelial Cells ; Humans ; Organoids

المؤلفون: Aalbers BL; Department of pulmonology, UMC Utrecht, The Netherlands. Electronic address: b.l.aalbers-2@umcutrecht.nl., Brunsveld JE; Department of pediatric pulmonology, UMC Utrecht, The Netherlands., van der Ent CK; Department of pediatric pulmonology, UMC Utrecht, The Netherlands., van den Eijnden JC; Department of pulmonology, UMC Utrecht, The Netherlands., Beekman JM; Department of pediatric pulmonology, UMC Utrecht, The Netherlands., Heijerman HGM; Department of pulmonology, UMC Utrecht, The Netherlands.

المصدر: Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society [J Cyst Fibros] 2022 Mar; Vol. 21 (2), pp. 254-257. Date of Electronic Publication: 2022 Jan 31.

نوع المنشور: Case Reports

بيانات الدورية: Publisher: Elsevier Country of Publication: Netherlands NLM ID: 101128966 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1873-5010 (Electronic) Linking ISSN: 15691993 NLM ISO Abbreviation: J Cyst Fibros Subsets: MEDLINE

مواضيع طبية MeSH: Compassionate Use Trials* , Cystic Fibrosis*/diagnosis , Cystic Fibrosis*/drug therapy , Cystic Fibrosis*/genetics, Aminophenols/therapeutic use ; Benzodioxoles/therapeutic use ; Colforsin/pharmacology ; Cystic Fibrosis Transmembrane Conductance Regulator/genetics ; Female ; Genotype ; Humans ; Middle Aged ; Mutation ; Organoids ; Rectum

المؤلفون: Geurts MH; Hubrecht Institute, Royal Netherlands Academy of Arts and Sciences (KNAW) and University Medical Center Utrecht, Utrecht, the Netherlands.; Oncode Institute, Hubrecht Institute, Utrecht, the Netherlands., de Poel E; Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, the Netherlands.; Regenerative Medicine Utrecht, University Medical Center, Utrecht University, Utrecht, the Netherlands., Pleguezuelos-Manzano C; Hubrecht Institute, Royal Netherlands Academy of Arts and Sciences (KNAW) and University Medical Center Utrecht, Utrecht, the Netherlands.; Oncode Institute, Hubrecht Institute, Utrecht, the Netherlands., Oka R; Oncode Institute,Princes Maxima Center, Utrecht, The Netherlands., Carrillo L; Hubrecht Institute, Royal Netherlands Academy of Arts and Sciences (KNAW) and University Medical Center Utrecht, Utrecht, the Netherlands.; Oncode Institute, Hubrecht Institute, Utrecht, the Netherlands., Andersson-Rolf A; Hubrecht Institute, Royal Netherlands Academy of Arts and Sciences (KNAW) and University Medical Center Utrecht, Utrecht, the Netherlands.; Oncode Institute, Hubrecht Institute, Utrecht, the Netherlands., Boretto M; Hubrecht Institute, Royal Netherlands Academy of Arts and Sciences (KNAW) and University Medical Center Utrecht, Utrecht, the Netherlands.; Oncode Institute, Hubrecht Institute, Utrecht, the Netherlands., Brunsveld JE; Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, the Netherlands.; Regenerative Medicine Utrecht, University Medical Center, Utrecht University, Utrecht, the Netherlands., van Boxtel R; Oncode Institute,Princes Maxima Center, Utrecht, The Netherlands., Beekman JM; Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, the Netherlands.; Regenerative Medicine Utrecht, University Medical Center, Utrecht University, Utrecht, the Netherlands., Clevers H; Hubrecht Institute, Royal Netherlands Academy of Arts and Sciences (KNAW) and University Medical Center Utrecht, Utrecht, the Netherlands h.clevers@hubrecht.eu.; Oncode Institute, Hubrecht Institute, Utrecht, the Netherlands.

المصدر: Life science alliance [Life Sci Alliance] 2021 Aug 09; Vol. 4 (10). Date of Electronic Publication: 2021 Aug 09 (Print Publication: 2021).

نوع المنشور: Journal Article; Research Support, Non-U.S. Gov't

بيانات الدورية: Publisher: Life Science Alliance, LLC Country of Publication: United States NLM ID: 101728869 Publication Model: Electronic-Print Cited Medium: Internet ISSN: 2575-1077 (Electronic) Linking ISSN: 25751077 NLM ISO Abbreviation: Life Sci Alliance Subsets: MEDLINE

مواضيع طبية MeSH: CRISPR-Cas Systems* , Gene Editing* , Mutation* , Organoids*, Cystic Fibrosis Transmembrane Conductance Regulator/*genetics , Oncogenes/*genetics, Alleles ; Amino Acid Substitution ; Animals ; Epithelial Cells/metabolism ; Genetic Therapy/methods ; Hepatocytes/metabolism ; Humans ; Sequence Analysis, DNA

المؤلفون: Geurts MH; Hubrecht Institute, Royal Netherlands Academy of Arts and Sciences (KNAW) and University Medical Center Utrecht, 3584 CT Utrecht, the Netherlands; Oncode Institute, Hubrecht Institute, 3584 CT Utrecht, the Netherlands., de Poel E; Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, 3584 EA Utrecht, the Netherlands; Regenerative Medicine Utrecht, University Medical Center, Utrecht University, 3584 CT Utrecht, the Netherlands., Amatngalim GD; Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, 3584 EA Utrecht, the Netherlands; Regenerative Medicine Utrecht, University Medical Center, Utrecht University, 3584 CT Utrecht, the Netherlands., Oka R; Princess Maxima Center, 3584 CS Utrecht, the Netherlands; Oncode Institute, Princess Maxima Center, 3584 CS Utrecht, the Netherlands., Meijers FM; Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, 3584 EA Utrecht, the Netherlands; Regenerative Medicine Utrecht, University Medical Center, Utrecht University, 3584 CT Utrecht, the Netherlands., Kruisselbrink E; Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, 3584 EA Utrecht, the Netherlands; Regenerative Medicine Utrecht, University Medical Center, Utrecht University, 3584 CT Utrecht, the Netherlands., van Mourik P; Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, 3584 EA Utrecht, the Netherlands., Berkers G; Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, 3584 EA Utrecht, the Netherlands., de Winter-de Groot KM; Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, 3584 EA Utrecht, the Netherlands., Michel S; Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, 3584 EA Utrecht, the Netherlands., Muilwijk D; Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, 3584 EA Utrecht, the Netherlands., Aalbers BL; Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, 3584 EA Utrecht, the Netherlands., Mullenders J; Hubrecht Organoid Technology, 3584 CM, Utrecht, the Netherlands., Boj SF; Hubrecht Organoid Technology, 3584 CM, Utrecht, the Netherlands., Suen SWF; Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, 3584 EA Utrecht, the Netherlands; Regenerative Medicine Utrecht, University Medical Center, Utrecht University, 3584 CT Utrecht, the Netherlands., Brunsveld JE; Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, 3584 EA Utrecht, the Netherlands; Regenerative Medicine Utrecht, University Medical Center, Utrecht University, 3584 CT Utrecht, the Netherlands., Janssens HM; Department of Pediatrics, division of Respiratory Medicine and Allergology, ErasmusMC-Sophia Children's Hospital, University Hospital Rotterdam, 3015 GD Rotterdam, the Netherlands., Mall MA; Department of Pediatric Pulmonology, Immunology and Critical Care Medicine, Charité-Universitätsmedizin Berlin, 13353 Berlin, Germany; Berlin Institute of Health (BIH), 10178 Berlin, Germany., Graeber SY; Department of Pediatric Pulmonology, Immunology and Critical Care Medicine, Charité-Universitätsmedizin Berlin, 13353 Berlin, Germany; Berlin Institute of Health (BIH), 10178 Berlin, Germany., van Boxtel R; Princess Maxima Center, 3584 CS Utrecht, the Netherlands; Oncode Institute, Princess Maxima Center, 3584 CS Utrecht, the Netherlands., van der Ent CK; Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, 3584 EA Utrecht, the Netherlands., Beekman JM; Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, 3584 EA Utrecht, the Netherlands; Regenerative Medicine Utrecht, University Medical Center, Utrecht University, 3584 CT Utrecht, the Netherlands. Electronic address: j.beekman@umcutrecht.nl., Clevers H; Hubrecht Institute, Royal Netherlands Academy of Arts and Sciences (KNAW) and University Medical Center Utrecht, 3584 CT Utrecht, the Netherlands; Oncode Institute, Hubrecht Institute, 3584 CT Utrecht, the Netherlands. Electronic address: h.clevers@hubrecht.eu.

المصدر: Cell stem cell [Cell Stem Cell] 2020 Apr 02; Vol. 26 (4), pp. 503-510.e7. Date of Electronic Publication: 2020 Feb 20.

نوع المنشور: Journal Article; Research Support, Non-U.S. Gov't

بيانات الدورية: Publisher: Cell Press Country of Publication: United States NLM ID: 101311472 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1875-9777 (Electronic) Linking ISSN: 18759777 NLM ISO Abbreviation: Cell Stem Cell Subsets: MEDLINE

مواضيع طبية MeSH: Clustered Regularly Interspaced Short Palindromic Repeats* , Cystic Fibrosis*/genetics, Adenine ; Biological Specimen Banks ; CRISPR-Associated Protein 9/genetics ; CRISPR-Cas Systems/genetics ; Codon, Nonsense ; Gene Editing ; Humans ; Organoids/metabolism