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In Vivo Functional Study of Disease-associated Rare Human Variants Using Drosophila
Published on: August 20, 2019
Podocyturia: why it may have added value in rare diseases
Maria Dolores Sanchez-Niño1, Maria Vanessa Perez-Gomez1, Lara Valiño-Rivas1
1IIS-Fundacion Jimenez Diaz, School of Medicine, Universidad Autonoma de Madrid; Fundacion Renal Iñigo Alvarez de Toledo-IRSIN and REDINREN, Madrid, Spain.
Abstract:
Fabry disease is an inherited lysosomal disease in which defects in the GLA gene lead to α-galactosidase-A deficiency, and accumulation of glycosphingolipids, including lyso-Gb3, a podocyte stressor. Therapy is available as enzyme replacement therapy and, for some patients, the chaperone migalastat. A key decision is when to start therapy, given its costs and potential impact on some aspects of quality of life. The decision is especially difficult in otherwise asymptomatic patients. A delayed start of therapy may allow kidney injury to progress subclinically up to the development of irreversible lesions. Non-invasive tools to monitor subclinical kidney injury are needed. One such tool may be assessment of podocyturia. In this issue of CKJ, [Trimarchi H, Canzonieri R, Costales-Collaguazo C et al. Early decrease in the podocalyxin to synaptopodin ratio in urinary Fabry podocytes. Clin Kidney J 2019; doi.org/10.1093/ckj/sfy053] report on podocyturia assessment in Fabry nephropathy. Specifically, they report that podocalyxin may be lost from detached urinary podocytes.
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