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

Autor: 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

Publikováno v: STAR Protocols, Vol 1, Iss 1, Pp 100019- (2020)

Summary: This protocol describes the isolation, handling, culture of, and experiments with human colon stem cell organoids in the context of cystic fibrosis (CF). In human colon organoids, the function of cystic fibrosis transmembrane conductance reg

Externí odkaz: https://doaj.org/article/fefd7bcad3ec46998e45178ebd7ffc13

Forskolin-induced organoid swelling is associated with long-term cystic fibrosis disease progression

Autor: Danya Muilwijk, Eyleen de Poel, Peter van Mourik, Sylvia W.F. Suen, Annelotte M. Vonk, Jesse E. Brunsveld, Evelien Kruisselbrink, Hugo Oppelaar, Marne C. Hagemeijer, Gitte Berkers, Karin M. de Winter-de Groot, Sabine Heida-Michel, Stephan R. Jans, Hannah van Panhuis, Menno M. van der Eerden, Renske van der Meer, Jolt Roukema, Edward Dompeling, Els J.M. Weersink, Gerard H. Koppelman, Robert Vries, Domenique D. Zomer-van Ommen, Marinus J.C. Eijkemans, Cornelis K. van der Ent, Jeffrey M. Beekman

Publikováno v: European Respiratory Journal, 60
European Respiratory Journal, 60, 2
European Respiratory Journal, 60(2):2100508. European Respiratory Society
European respiratory journal, 60(2):2100508. European Respiratory Society
Muilwijk, D, de Poel, E, van Mourik, P, Suen, S W F, Vonk, A M, Brunsveld, J E, Kruisselbrink, E, Oppelaar, H, Hagemeijer, M C, Berkers, G, de Winter-de Groot, K M, Heida-Michel, S, Jans, S R, van Panhuis, H, van der Eerden, M M, van der Meer, R, Roukema, J, Dompeling, E, Weersink, E J M, Koppelman, G H, Vries, R, Zomer-van Ommen, D D, Eijkemans, M J C, van der Ent, C K & Beekman, J M 2022, ' Forskolin-induced organoid swelling is associated with long-term cystic fibrosis disease progression ', European Respiratory Journal, vol. 60, no. 2, 2100508 . https://doi.org/10.1183/13993003.00508-2021

RationaleCystic fibrosis (CF) is a monogenic life-shortening disease associated with highly variable individual disease progression which is difficult to predict. Here we assessed the association of forskolin-induced swelling (FIS) of patient-derived

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::2cf9da16f7bb9568249c8cf57a8787cc
https://pubmed.ncbi.nlm.nih.gov/35086832

Functional Restoration of CFTR Nonsense Mutations in Intestinal Organoids

Autor: E. de Poel, Els J.M. Weersink, Simon Y. Graeber, Marcus A. Mall, Gerard H. Koppelman, C.K. van der Ent, Sylvia W.F. Suen, Evelien Kruisselbrink, M M van der Eerden, Sacha Spelier, Jeffrey M. Beekman

Publikováno v: Journal of Cystic Fibrosis, 21(2), 246-253. ELSEVIER SCIENCE BV
Journal of cystic fibrosis. Elsevier
Journal of Cystic Fibrosis, 21(2), 246-253. Elsevier

BACKGROUND: Pharmacotherapies for people with cystic fibrosis (pwCF) who have premature termination codons (PTCs) in the cystic fibrosis transmembrane conductance regulator (CFTR) gene are under development. Thus far, clinical studies focused on comp

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::63ab935aa2e53101e6c41ada3b19e696
http://www.scopus.com/inward/record.url?scp=85117191131&partnerID=8YFLogxK

CRISPR-Based Adenine Editors Correct Nonsense Mutations in a Cystic Fibrosis Organoid Biobank

Autor: Gimano D. Amatngalim, Ruben van Boxtel, Sabine Michel, Bente L. Aalbers, Cornelis K. van der Ent, Karin M. de Winter-de Groot, Rurika Oka, Maarten H. Geurts, Eyleen de Poel, D. Muilwijk, Evelien Kruisselbrink, Hettie M. Janssens, Simon Y. Graeber, Sylvia W.F. Suen, Marcus A. Mall, Sylvia F. Boj, Gitte Berkers, Fleur M. Meijers, Jeffrey M. Beekman, Jesse E. Brunsveld, Jasper Mullenders, Peter van Mourik, Hans Clevers

Publikováno v: Cell Stem Cell, 26(4), 503-510.e7. Cell Press
Cell Stem Cell, 26(4), 503-510. Cell Press

Adenine base editing (ABE) enables enzymatic conversion from A-T into G-C base pairs. ABE holds promise for clinical application, as it does not depend on the introduction of double-strand breaks, contrary to conventional CRISPR/Cas9-mediated genome

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::beb71bab9b7ebab5ed7f8188f09953f5
https://pure.knaw.nl/portal/en/publications/bee72aae-e110-43f9-bd4f-c4419015f333

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

Autor: C. de Boeck, Margarida D. Amaral, Jeffrey M. Beekman, Anabela S. Ramalho, Sylvia W.F. Suen, Annelotte M. Vonk, K. van der Ent, J. Pott, François Vermeulen, M. Bierlaagh, Iris A.L. Silva

Publikováno v: Journal of Cystic Fibrosis. 20:S17

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::1a3a242114164debdf2632b63dd0d798
https://doi.org/10.1016/s1569-1993(21)00963-2