Characterizing responses to CFTR-modulating drugs using rectal organoids derived from subjects with cystic fibrosis-Reference-Cited by-同舟云学术

Characterizing responses to CFTR-modulating drugs using rectal organoids derived from subjects with cystic fibrosis

Published:2016-06-22 Issue:344 Volume:8 Page:
ISSN:1946-6234
Container-title:Science Translational Medicine
language:en
Short-container-title:Sci. Transl. Med.

Author:

Dekkers Johanna F.¹²,Berkers Gitte¹,Kruisselbrink Evelien¹²,Vonk Annelotte¹²,de Jonge Hugo R.³,Janssens Hettie M.⁴,Bronsveld Inez⁵,van de Graaf Eduard A.⁵,Nieuwenhuis Edward E. S.⁶,Houwen Roderick H. J.⁶,Vleggaar Frank P.⁷,Escher Johanna C.⁸,de Rijke Yolanda B.⁹,Majoor Christof J.¹⁰,Heijerman Harry G. M.¹¹,de Winter–de Groot Karin M.¹,Clevers Hans¹²,van der Ent Cornelis K.¹,Beekman Jeffrey M.¹²¹³

Affiliation:

1. Department of Pediatric Pulmonology, Wilhelmina Children’s Hospital, University Medical Center Utrecht, 3584 EA Utrecht, Netherlands.

2. Laboratory of Translational Immunology, Wilhelmina Children’s Hospital, University Medical Center Utrecht, 3584 EA Utrecht, Netherlands.

3. Department of Gastroenterology and Hepatology, Erasmus University Medical Center, 3015 CE Rotterdam, Netherlands.

4. Department of Pediatric Pulmonology, Erasmus University Medical Center/Sophia Children’s Hospital, 3015 CN Rotterdam, Netherlands.

5. Department of Pulmonology, University Medical Center Utrecht, 3584 CX Utrecht, Netherlands.

6. Department of Pediatric Gastroenterology, Wilhelmina Children’s Hospital, University Medical Center Utrecht, 3584 EA Utrecht, Netherlands.

7. Department of Gastroenterology and Hepatology, University Medical Center Utrecht, 3584 CX Utrecht, Netherlands.

8. Department of Pediatric Gastroenterology, Erasmus University Medical Center/Sophia Children’s Hospital, 3015 CN Rotterdam, Netherlands.

9. Department of Clinical Chemistry, Erasmus University Medical Center/Sophia Children’s Hospital, 3015 CN Rotterdam, Netherlands.

10. Department of Respiratory Medicine, Academic Medical Center, 1105 AZ Amsterdam, Netherlands.

11. Department of Pulmonology and Cystic Fibrosis, Haga Teaching Hospital, 2545 CH The Hague, Netherlands.

12. Hubrecht Institute for Developmental Biology and Stem Cell Research and University Medical Center Utrecht, 3584 CT Utrecht, Netherlands.

13. Regenerative Medicine Center Utrecht, University Medical Center Utrecht, 3584 CX Utrecht, Netherlands.

Abstract

Rectal organoids from subjects with cystic fibrosis can be used to assess responses to drugs that modulate CFTR.

Funder

Dutch Cystic Fibrosis Foundation

Wilhelmina Children's Hospital Foundation

Dutch Health Organization ZonMw, Netherlands

Publisher

American Association for the Advancement of Science (AAAS)

Subject

General Medicine

Reference44 articles.

1. New pharmacological approaches for cystic fibrosis: Promises, progress, pitfalls

2. Defining the disease liability of variants in the cystic fibrosis transmembrane conductance regulator gene

3. Cystic fibrosis genetics: from molecular understanding to clinical application

4. A CFTR Potentiator in Patients with Cystic Fibrosis and theG551DMutation

5. Effect of VX-770 in Persons with Cystic Fibrosis and the G551D-CFTRMutation

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