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

Rezultaty

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

Publikacje

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

Autorzy: Iris A. L. Silva, Aires Duarte, Fernando A. L. Marson, Raquel Centeio, Tereza Doušová, Karl Kunzelmann, Margarida D. Amaral
Opublikowane w: Frontiers in Physiology, Numer 11, 2020, ISSN 1664-042X
Wydawca: Frontiers Research Foundation
DOI: 10.3389/fphys.2020.604580

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

Autorzy: Michael A. Lensink, Karin R. Jongsma, Sarah N. Boers, Jacquelien J. Noordhoek, Jeffrey M. Beekman, Annelien L. Bredenoord
Opublikowane w: Development, Numer 147/7, 2020, ISSN 0950-1991
Wydawca: 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

Autorzy: Michael A. Lensink, Sarah N. Boers, Karin R. Jongsma, Sarah E. Carter, Cornelis K. van der Ent, Annelien L. Bredenoord
Opublikowane w: Journal of Cystic Fibrosis, Numer 20/3, 2021, Strona(/y) 443-451, ISSN 1569-1993
Wydawca: 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

Autorzy: 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
Opublikowane w: Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease, Numer 1866/11, 2020, Strona(/y) 165905, ISSN 0925-4439
Wydawca: 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

Autorzy: Michael A. Lensink, Karin R. Jongsma, Sarah N. Boers, Annelien L. Bredenoord
Opublikowane w: BMC Medical Ethics, Numer 23, 2022, ISSN 1472-6939
Wydawca: BioMed Central
DOI: 10.1186/s12910-022-00823-7

Intestinal organoids to model cystic fibrosis

Autorzy: Peter van Mourik, Jeffrey M. Beekman, Cornelis K. van der Ent
Opublikowane w: European Respiratory Journal, Numer 54/1, 2019, Strona(/y) 1802379, ISSN 0903-1936
Wydawca: European Respiratory Society
DOI: 10.1183/13993003.02379-2018

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

Autorzy: 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
Opublikowane w: Life Science Alliance, Numer 5, 2024, Strona(/y) e202101320, ISSN 2575-1077
Wydawca: 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

Autorzy: 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
Opublikowane w: STAR Protocols, Numer 1/1, 2020, Strona(/y) 100019, ISSN 2666-1667
Wydawca: 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

Autorzy: 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
Opublikowane w: International Journal of Molecular Sciences, Numer 24, 2023, Strona(/y) 14539, ISSN 1422-0067
Wydawca: 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

Autorzy: 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
Opublikowane w: Journal of Cystic Fibrosis, Numer 20/3, 2021, Strona(/y) 473-484, ISSN 1569-1993
Wydawca: Elsevier BV
DOI: 10.1016/j.jcf.2020.08.007

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

Autorzy: 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
Opublikowane w: Journal of Cystic Fibrosis, Numer 20, 2023, Strona(/y) S17, ISSN 1569-1993
Wydawca: 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

Autorzy: 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
Opublikowane w: Translational Medicine Communications, Numer 5/1, 2020, ISSN 2396-832X
Wydawca: 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

Autorzy: Iris A. L. Silva, Violeta Railean, Aires Duarte, Margarida D. Amaral
Opublikowane w: Journal of Personalized Medicine, Numer 11/5, 2021, Strona(/y) 421, ISSN 2075-4426
Wydawca: MDPI
DOI: 10.3390/jpm11050421

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