Personalised Treatment For Cystic Fibrosis Patients With Ultra-rare CFTR Mutations (and beyond)

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A suite of branded template material and a website

A suite of branded template material and a website for dissemination and public awareness of the HIT-CF project

Publications

Assessment of Distinct Electrophysiological Parameters in Rectal Biopsies for the Choice of the Best Diagnosis/Prognosis Biomarkers for Cystic Fibrosis

Auteurs: Iris A. L. Silva, Aires Duarte, Fernando A. L. Marson, Raquel Centeio, Tereza Doušová, Karl Kunzelmann, Margarida D. Amaral
Publié dans: Frontiers in Physiology, Numéro 11, 2020, ISSN 1664-042X
Éditeur: Frontiers Research Foundation
DOI: 10.3389/fphys.2020.604580

Centralized intestinal organoid generation is a feasible and safe approach for personalized medicine as demonstrated in the HIT-CF Europe Organoid Study

Auteurs: Marlou C. Bierlaagh, Peter van Mourik, Annelotte M. Vonk, Johanna Pott, Danya Muilwijk, Gitte Berkers, Bente L. Aalbers, Frank P. Vleggaar, Sabine Michel, Sylvia F. Boj, Robert G.J. Vries, Jeffrey M. Beekman, Cornelis K. van der Ent
Publié dans: Journal of Cystic Fibrosis, 2024, ISSN 1569-1993
Éditeur: Elsevier BV
DOI: 10.1016/j.jcf.2024.04.016

Responsible use of organoids in precision medicine: the need for active participant involvement

Auteurs: Michael A. Lensink, Karin R. Jongsma, Sarah N. Boers, Jacquelien J. Noordhoek, Jeffrey M. Beekman, Annelien L. Bredenoord
Publié dans: Development, Numéro 147/7, 2020, ISSN 0950-1991
Éditeur: The Company of Biologists Ltd.
DOI: 10.1242/dev.177972

Organoids for personalized treatment of Cystic Fibrosis: Professional perspectives on the ethics and governance of organoid biobanking

Auteurs: Michael A. Lensink, Sarah N. Boers, Karin R. Jongsma, Sarah E. Carter, Cornelis K. van der Ent, Annelien L. Bredenoord
Publié dans: Journal of Cystic Fibrosis, Numéro 20/3, 2021, Page(s) 443-451, ISSN 1569-1993
Éditeur: Elsevier BV
DOI: 10.1016/j.jcf.2020.11.015

Organoids as a personalized medicine tool for ultra-rare mutations in cystic fibrosis: The case of S955P and 1717-2A>G

Auteurs: Iris A.L. Silva, Tereza Doušová, Sofia Ramalho, Raquel Centeio, Luka A. Clarke, Violeta Railean, Hugo M. Botelho, Andrea Holubová, Iveta Valášková, Jiunn-Tyng Yeh, Tzyh-Chang Hwang, Carlos M. Farinha, Karl Kunzelmann, Margarida D. Amaral
Publié dans: Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease, Numéro 1866/11, 2020, Page(s) 165905, ISSN 0925-4439
Éditeur: Elsevier BV
DOI: 10.1016/j.bbadis.2020.165905

Better governance starts with better words: why responsible human tissue research demands a change of language

Auteurs: Michael A. Lensink, Karin R. Jongsma, Sarah N. Boers, Annelien L. Bredenoord
Publié dans: BMC Medical Ethics, Numéro 23, 2022, ISSN 1472-6939
Éditeur: BioMed Central
DOI: 10.1186/s12910-022-00823-7

Intestinal organoids to model cystic fibrosis

Auteurs: Peter van Mourik, Jeffrey M. Beekman, Cornelis K. van der Ent
Publié dans: European Respiratory Journal, Numéro 54/1, 2019, Page(s) 1802379, ISSN 0903-1936
Éditeur: European Respiratory Society
DOI: 10.1183/13993003.02379-2018

Measuring cystic fibrosis drug responses in organoids derived from 2D differentiated nasal epithelia

Auteurs: Gimano D Amatngalim, Lisa W Rodenburg, Bente L Aalbers, Henriette HM Raeven, Ellen M Aarts, Dounia Sarhane, Sacha Spelier, Juliet W Lefferts, Iris AL Silva, Wilco Nijenhuis, Sacha Vrendenbarg, Evelien Kruisselbrink, Jesse E Brunsveld, Cornelis M van Drunen, Sabine Michel, Karin M de Winter-de Groot, Harry G Heijerman, Lukas C Kapitein, Magarida D Amaral, Cornelis K van der Ent, Jeffrey M Beekman
Publié dans: Life Science Alliance, Numéro 5, 2024, Page(s) e202101320, ISSN 2575-1077
Éditeur: https://www.life-science-alliance.org/
DOI: 10.26508/lsa.202101320

Protocol for Application, Standardization and Validation of the Forskolin-Induced Swelling Assay in Cystic Fibrosis Human Colon Organoids

Auteurs: Annelotte M. Vonk, Peter van Mourik, Anabela S. Ramalho, Iris A.L. Silva, Marvin Statia, Evelien Kruisselbrink, Sylvia W.F. Suen, Johanna F. Dekkers, Frank P. Vleggaar, Roderick H.J. Houwen, Jasper Mullenders, Sylvia F. Boj, Robert Vries, Margarida D. Amaral, Kris de Boeck, Cornelis K. van der Ent, Jeffrey M. Beekman
Publié dans: STAR Protocols, Numéro 1/1, 2020, Page(s) 100019, ISSN 2666-1667
Éditeur: Cell Press
DOI: 10.1016/j.xpro.2020.100019

CFTR Function Restoration upon Elexacaftor/Tezacaftor/Ivacaftor Treatment in Patient-Derived Intestinal Organoids with Rare CFTR Genotypes

Auteurs: Juliet W. Lefferts, Marlou C. Bierlaagh, Suzanne Kroes, Natascha D. A. Nieuwenhuijze, Heleen N. Sonneveld van Kooten, Paul J. Niemöller, Tibo F. Verburg, Hettie M. Janssens, Danya Muilwijk, Sam F. B. van Beuningen, Cornelis K. van der Ent, Jeffrey M. Beekman
Publié dans: International Journal of Molecular Sciences, Numéro 24, 2023, Page(s) 14539, ISSN 1422-0067
Éditeur: Multidisciplinary Digital Publishing Institute (MDPI)
DOI: 10.3390/ijms241914539

Extensive CFTR sequencing through NGS in Brazilian individuals with cystic fibrosis: unravelling regional discrepancies in the country

Auteurs: Luiz Vicente Ribeiro Ferreira da Silva Filho, Paulo José Cauduro Maróstica, Rodrigo Abensur Athanazio, Francisco José Caldeira Reis, Neiva Damaceno, Angela Tavares Paes, Adilson Yuuji Hira, David Schlesinger, Fernando Kok, Margarida D. Amaral, Mara Lícia Machado Antunes, Lilian Cristina Ferreira Andries, Virginia Auxiliadora Freitas de Castro, Fabíola Villac Adde, Maria Fernanda Botelho Herna
Publié dans: Journal of Cystic Fibrosis, Numéro 20/3, 2021, Page(s) 473-484, ISSN 1569-1993
Éditeur: Elsevier BV
DOI: 10.1016/j.jcf.2020.08.007

Repeatability and reproducibility of the Forskolin-induced swelling (FIS) assay on intestinal organoids from people with Cystic Fibrosis

Auteurs: Marlou C. Bierlaagh, Anabela S. Ramalho, Iris A.L. Silva, Annelotte M. Vonk, Rutger M. van den Bor, Peter van Mourik, Johanna Pott, Sylvia W.F. Suen, Sylvia F. Boj, Robert G.J. Vries, Elise Lammertyn, François Vermeulen, Margarida D. Amaral, Kris de Boeck, Cornelis K. van der Ent, Jeffrey M. Beekman
Publié dans: Journal of Cystic Fibrosis, 2024, ISSN 1569-1993
Éditeur: Elsevier BV
DOI: 10.1016/j.jcf.2024.04.014

WS09.1 First results of the HIT-CF ex vivo organoid study show rescue of CFTR with ultra-rare mutations by a novel triple combination of CFTR modulators

Auteurs: I.A.L. Silva, A.S. Ramalho, A.M. Vonk, M. Bierlaagh, S.W.F. Suen, J. Pott, M.D. Amaral, F. Vermeulen, C. De Boeck, J.M. Beekman, K. van der Ent
Publié dans: Journal of Cystic Fibrosis, Numéro 20, 2023, Page(s) S17, ISSN 1569-1993
Éditeur: Elsevier BV
DOI: 10.1016/s1569-1993(21)00963-2

Rationale and design of the HIT-CF organoid study: stratifying cystic fibrosis patients based on intestinal organoid response to different CFTR-modulators

Auteurs: Peter van Mourik, Sabine Michel, Annelotte M. Vonk, Jeffrey M. Beekman, Cornelis K. van der Ent, Hilde De Keyser, Elise Lammertyn, Silke van Koningsbruggen-Rietschel, Lutz Naehrlich, Judith Pool, Marc van de Craen, Begoña Aguilera, Johanna Pott, Rob G. J. Vries, Sylvia F. Boj, Kris De Boeck, François Vermeulen, Anabela S. Ramalho, Iris A. L. Silva, Margarida D. Amaral, Matthew Goddeeris, Geoffre
Publié dans: Translational Medicine Communications, Numéro 5/1, 2020, ISSN 2396-832X
Éditeur: BMC - part of springer Nature
DOI: 10.1186/s41231-020-00060-3

Personalized Medicine Based on Nasal Epithelial Cells: Comparative Studies with Rectal Biopsies and Intestinal Organoids

Auteurs: Iris A. L. Silva, Violeta Railean, Aires Duarte, Margarida D. Amaral
Publié dans: Journal of Personalized Medicine, Numéro 11/5, 2021, Page(s) 421, ISSN 2075-4426
Éditeur: MDPI
DOI: 10.3390/jpm11050421

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