Bile Acid Metabolism Changes in Patients with a CRB1-Associated Inherited Retinal Degeneration

Purpose: To compare the plasma metabolic profile of patients with a Crumbs homolog 1-associated inherited retinal degeneration (CRB1-IRD) with that of healthy controls (HCs). Design: A case-control study. Participants: A cohort of 30 Dutch patients with CRB1-IRD and 29 Dutch HCs. Methods: The MxP Qu...

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Main Authors: Lude Moekotte, MD, PhD, Joke H. de Boer, MD, PhD, Sanne Hiddingh, MSc, Bram Gerritsen, PhD, Jutta Lintelmann, PhD, Alexander Cecil, PhD, L. Ingeborgh van den Born, MD, PhD, Xuan-Thanh-An Nguyen, MD, PhD, Camiel J.F. Boon, MD, PhD, Maria M. van Genderen, MD, PhD, Jonas J.W. Kuiper, PhD
Format: Article
Language:English
Published: Elsevier 2025-07-01
Series:Ophthalmology Science
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Online Access:http://www.sciencedirect.com/science/article/pii/S2666914525000028
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author Lude Moekotte, MD, PhD
Joke H. de Boer, MD, PhD
Sanne Hiddingh, MSc
Bram Gerritsen, PhD
Jutta Lintelmann, PhD
Alexander Cecil, PhD
L. Ingeborgh van den Born, MD, PhD
Xuan-Thanh-An Nguyen, MD, PhD
Camiel J.F. Boon, MD, PhD
Maria M. van Genderen, MD, PhD
Jonas J.W. Kuiper, PhD
author_facet Lude Moekotte, MD, PhD
Joke H. de Boer, MD, PhD
Sanne Hiddingh, MSc
Bram Gerritsen, PhD
Jutta Lintelmann, PhD
Alexander Cecil, PhD
L. Ingeborgh van den Born, MD, PhD
Xuan-Thanh-An Nguyen, MD, PhD
Camiel J.F. Boon, MD, PhD
Maria M. van Genderen, MD, PhD
Jonas J.W. Kuiper, PhD
author_sort Lude Moekotte, MD, PhD
collection DOAJ
description Purpose: To compare the plasma metabolic profile of patients with a Crumbs homolog 1-associated inherited retinal degeneration (CRB1-IRD) with that of healthy controls (HCs). Design: A case-control study. Participants: A cohort of 30 Dutch patients with CRB1-IRD and 29 Dutch HCs. Methods: The MxP Quant 500 Kit was used for measuring metabolite concentrations. We fitted a linear regression model with adjustments for age and sex based on the concentration of metabolites in micromolar (micromoles per liter) or on the sums and ratios of metabolites to determine differences between patients and controls. Main Outcome Measures: Plasma concentration of 619 metabolites. Results: Overrepresentation of pathways among metabolites associated strongest to CRB1-IRDs (P < 0.05, n = 62) identified amino acid pathways (such as β-alanine, histidine, and glycine/serine) and bile acid biosynthesis, driven by a decrease in deoxycholic acid derivatives produced by gut microbiota. Enrichment analysis of metabolic classes across the plasma metabolic profile further identified significant positive enrichment for lipid metabolites glycerophospholipids, cholesterol esters, and ceramides, and significant depletion for bile acid metabolites. Further investigation of the sums and ratios (i.e., metabolism indicators) ascertained a significant decrease in intestinal microbial-dependent secondary bile acid classes. Conclusions: Lipid metabolic alterations and decreased microbiota-related secondary bile acid concentrations indicate significant alterations in gut metabolism in patients with a CRB1-IRD. Financial Disclosure(s): Proprietary or commercial disclosure may be found in the Footnotes and Disclosures at the end of this article.
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spelling doaj-art-86f4d2e51474479685389822fdaf06cc2025-08-20T02:12:14ZengElsevierOphthalmology Science2666-91452025-07-015410070410.1016/j.xops.2025.100704Bile Acid Metabolism Changes in Patients with a CRB1-Associated Inherited Retinal DegenerationLude Moekotte, MD, PhD0Joke H. de Boer, MD, PhD1Sanne Hiddingh, MSc2Bram Gerritsen, PhD3Jutta Lintelmann, PhD4Alexander Cecil, PhD5L. Ingeborgh van den Born, MD, PhD6Xuan-Thanh-An Nguyen, MD, PhD7Camiel J.F. Boon, MD, PhD8Maria M. van Genderen, MD, PhD9Jonas J.W. Kuiper, PhD10Department of Ophthalmology, University Medical Center Utrecht, Utrecht, The Netherlands; Correspondence: Lude Moekotte, MD, PhD, Department of Ophthalmology, University Medical Center Utrecht, Heidelberglaan 100, Utrecht 3584 CX, the Netherlands.Department of Ophthalmology, University Medical Center Utrecht, Utrecht, The NetherlandsDepartment of Ophthalmology, University Medical Center Utrecht, Utrecht, The NetherlandsDepartment of Ophthalmology, University Medical Center Utrecht, Utrecht, The NetherlandsMetabolomics and Proteomics Core (MPC), Helmholtz Zentrum München, Munich, GermanyMetabolomics and Proteomics Core (MPC), Helmholtz Zentrum München, Munich, GermanyThe Rotterdam Eye Hospital, Rotterdam, The NetherlandsDepartment of Ophthalmology, Leiden University Medical Center, Leiden, The NetherlandsDepartment of Ophthalmology, Leiden University Medical Center, Leiden, The Netherlands; Department of Ophthalmology, Amsterdam University Medical Center, Amsterdam, The NetherlandsDepartment of Ophthalmology, University Medical Center Utrecht, Utrecht, The Netherlands; Bartiméus, Diagnostic Centre for Complex Visual Disorders, Zeist, The NetherlandsDepartment of Ophthalmology, University Medical Center Utrecht, Utrecht, The NetherlandsPurpose: To compare the plasma metabolic profile of patients with a Crumbs homolog 1-associated inherited retinal degeneration (CRB1-IRD) with that of healthy controls (HCs). Design: A case-control study. Participants: A cohort of 30 Dutch patients with CRB1-IRD and 29 Dutch HCs. Methods: The MxP Quant 500 Kit was used for measuring metabolite concentrations. We fitted a linear regression model with adjustments for age and sex based on the concentration of metabolites in micromolar (micromoles per liter) or on the sums and ratios of metabolites to determine differences between patients and controls. Main Outcome Measures: Plasma concentration of 619 metabolites. Results: Overrepresentation of pathways among metabolites associated strongest to CRB1-IRDs (P < 0.05, n = 62) identified amino acid pathways (such as β-alanine, histidine, and glycine/serine) and bile acid biosynthesis, driven by a decrease in deoxycholic acid derivatives produced by gut microbiota. Enrichment analysis of metabolic classes across the plasma metabolic profile further identified significant positive enrichment for lipid metabolites glycerophospholipids, cholesterol esters, and ceramides, and significant depletion for bile acid metabolites. Further investigation of the sums and ratios (i.e., metabolism indicators) ascertained a significant decrease in intestinal microbial-dependent secondary bile acid classes. Conclusions: Lipid metabolic alterations and decreased microbiota-related secondary bile acid concentrations indicate significant alterations in gut metabolism in patients with a CRB1-IRD. Financial Disclosure(s): Proprietary or commercial disclosure may be found in the Footnotes and Disclosures at the end of this article.http://www.sciencedirect.com/science/article/pii/S2666914525000028Bile acidsCRB1Inherited retinal degenerationsMetabolomics
spellingShingle Lude Moekotte, MD, PhD
Joke H. de Boer, MD, PhD
Sanne Hiddingh, MSc
Bram Gerritsen, PhD
Jutta Lintelmann, PhD
Alexander Cecil, PhD
L. Ingeborgh van den Born, MD, PhD
Xuan-Thanh-An Nguyen, MD, PhD
Camiel J.F. Boon, MD, PhD
Maria M. van Genderen, MD, PhD
Jonas J.W. Kuiper, PhD
Bile Acid Metabolism Changes in Patients with a CRB1-Associated Inherited Retinal Degeneration
Ophthalmology Science
Bile acids
CRB1
Inherited retinal degenerations
Metabolomics
title Bile Acid Metabolism Changes in Patients with a CRB1-Associated Inherited Retinal Degeneration
title_full Bile Acid Metabolism Changes in Patients with a CRB1-Associated Inherited Retinal Degeneration
title_fullStr Bile Acid Metabolism Changes in Patients with a CRB1-Associated Inherited Retinal Degeneration
title_full_unstemmed Bile Acid Metabolism Changes in Patients with a CRB1-Associated Inherited Retinal Degeneration
title_short Bile Acid Metabolism Changes in Patients with a CRB1-Associated Inherited Retinal Degeneration
title_sort bile acid metabolism changes in patients with a crb1 associated inherited retinal degeneration
topic Bile acids
CRB1
Inherited retinal degenerations
Metabolomics
url http://www.sciencedirect.com/science/article/pii/S2666914525000028
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