Literature DB >> 31718986

Pathogenesis of Fabry nephropathy: The pathways leading to fibrosis.

Paula Adriana Rozenfeld1, María de Los Angeles Bolla2, Pedro Quieto3, Antonio Pisani4, Sandro Feriozzi5, Pablo Neuman6, Constanza Bondar7.   

Abstract

BACKGROUND: Kidney is one of the main target organs in Fabry disease, a lysosomal X-linked genetic disorder. Renal involvement is characterized by proteinuria and progressive chronic kidney disease leading to end-stage renal disease. Pathogenic mechanisms in the progression of renal damage in Fabry disease are not thoroughly known yet. The lysosomal Gb3 deposition is the first step of complex pathological pathways resulting in renal sclerosis/fibrosis. Our hypothesis is that Fabry disease associated cellular alterations in tubular cells induce the production of TGF-β1, which mediate transdifferentiation of renal cells into myofibroblasts resulting in fibrosis of renal tissue.
OBJECTIVES: The aim of this work is to study the mechanisms leading to fibrosis in kidney from human Fabry patients.
METHODS: Fifteen renal biopsies from naïve Fabry patients were included. Histological and immunohistochemical analysis was carried out.
RESULTS: Positive staining for TGF-β1 was found in tubular epithelial cells in biopsies from Fabry patients. Apoptosis was determined by active caspase 3 staining in tubular and mesangial glomerular cells. Due to TGF-β1 is the main profibrotic cytokine and induces accumulation of myofibroblasts, we performed a study for its marker α-smooth muscle actin (α-SMA). This study revealed expression of α-SMA on pericytes surrounding peritubular capillaries, smooth muscle cells of blood vessels, mesangial cells and periglomerular zone. TGF-β1 is produced mainly by tubular cells in Fabry kidney biopsies probably inducing cellular trans-differentiation of renal cells into myofibroblasts. A positive staining for a marker of myofibroblasts was present, affirming the presence of those profibrotic cells.
CONCLUSIONS: These results show for the first time that TGF-β1 is expressed in human renal tissue from Fabry patients, and that this profibrotic cytokine is mainly produced by proximal tubular cells. In addition both, peritubular interstitium and glomeruli, presented cells positive for myofibroblasts markers.
Copyright © 2019 Elsevier Inc. All rights reserved.

Entities:  

Keywords:  Fabry; Fibrosis; Kidney biopsy; Nephropathy; TGF-β

Year:  2019        PMID: 31718986     DOI: 10.1016/j.ymgme.2019.10.010

Source DB:  PubMed          Journal:  Mol Genet Metab        ISSN: 1096-7192            Impact factor:   4.797


  8 in total

1.  Fabry disease - a genetically conditioned extremely rare disease with a very unusual course.

Authors:  Mirosław Śnit; Marcela Przyłudzka; Władysław Grzeszczak
Journal:  Intractable Rare Dis Res       Date:  2022-02

2.  Long-term follow-up of renal function in patients treated with migalastat for Fabry disease.

Authors:  Daniel G Bichet; Roser Torra; Eric Wallace; Derralynn Hughes; Roberto Giugliani; Nina Skuban; Eva Krusinska; Ulla Feldt-Rasmussen; Raphael Schiffmann; Kathy Nicholls
Journal:  Mol Genet Metab Rep       Date:  2021-08-04

Review 3.  Cardiovascular magnetic resonance native T1 mapping in Anderson-Fabry disease: a systematic review and meta-analysis.

Authors:  Andrea Ponsiglione; Michele Gambardella; Roberta Green; Valeria Cantoni; Carmela Nappi; Raffaele Ascione; Marco De Giorgi; Renato Cuocolo; Antonio Pisani; Mario Petretta; Alberto Cuocolo; Massimo Imbriaco
Journal:  J Cardiovasc Magn Reson       Date:  2022-05-23       Impact factor: 6.903

4.  Brazilian consensus recommendations for the diagnosis, screening, and treatment of individuals with fabry disease: Committee for Rare Diseases - Brazilian Society of Nephrology/2021.

Authors:  Cassiano Augusto Braga Silva; Luis Gustavo Modelli de Andrade; Maria Helena Vaisbich; Fellype de Carvalho Barreto
Journal:  J Bras Nefrol       Date:  2022 Apr-Jun

5.  Fabry disease patients have an increased risk of stroke in the COVID-19 ERA. A hypothesis.

Authors:  R C Reisin; P Rozenfeld; P Bonardo
Journal:  Med Hypotheses       Date:  2020-09-17       Impact factor: 1.538

Review 6.  Renal Manifestations of Fabry Disease: A Narrative Review.

Authors:  Cassiano Augusto Braga Silva; José A Moura-Neto; Marlene Antônia Dos Reis; Osvaldo Merege Vieira Neto; Fellype Carvalho Barreto
Journal:  Can J Kidney Health Dis       Date:  2021-01-19

7.  α-Galactosidase a Deficiency in Fabry Disease Leads to Extensive Dysregulated Cellular Signaling Pathways in Human Podocytes.

Authors:  Ulrich Jehn; Samet Bayraktar; Solvey Pollmann; Veerle Van Marck; Thomas Weide; Hermann Pavenstädt; Eva Brand; Malte Lenders
Journal:  Int J Mol Sci       Date:  2021-10-20       Impact factor: 5.923

8.  Dysregulated DNA methylation in the pathogenesis of Fabry disease.

Authors:  Jin-Song Shen; Uthra Balaji; Kunitoshi Shigeyasu; Yoshinaga Okugawa; Siamak Jabbarzadeh-Tabrizi; Taniqua S Day; Erland Arning; John Marshall; Seng H Cheng; Jinghua Gu; Raphael Schiffmann; Teodoro Bottiglieri; Ajay Goel
Journal:  Mol Genet Metab Rep       Date:  2022-09-26
  8 in total

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