Hematopoietic Stem Cells Transplantation Can Normalize Thyroid Function in a Cystinosis Mouse Model

Endocrinology
H P Gaide ChevronnayPierre J Courtoy

Abstract

Hypothyroidism is the most frequent and earliest endocrine complication in cystinosis, a multisystemic lysosomal storage disease caused by defective transmembrane cystine transporter, cystinosin (CTNS gene). We recently demonstrated in Ctns(-/-) mice that altered thyroglobulin biosynthesis associated with endoplasmic reticulum stress, combined with defective lysosomal processing, caused hypothyroidism. In Ctns(-/-) kidney, hematopoietic stem cell (HSC) transplantation provides long-term functional and structural protection. Tissue repair involves transfer of cystinosin-bearing lysosomes from HSCs differentiated as F4/80 macrophages into deficient kidney tubular cells, via tunneling nanotubes that cross basement laminae. Here we evaluated the benefit of HSC transplantation for cystinotic thyroid and investigated the underlying mechanisms. HSC engraftment in Ctns(-/-) thyroid drastically decreased cystine accumulation, normalized the TSH level, and corrected the structure of a large fraction of thyrocytes. In the thyroid microenvironment, HSCs differentiated into a distinct, mixed macrophage/dendritic cell lineage expressing CD45 and major histocompatibility complex II but low CD11b and F4/80. Grafted HSCs closely apposed to foll...Continue Reading

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Citations

Dec 19, 2016·Nature Reviews. Nephrology·Stephanie Cherqui, Pierre J Courtoy
Dec 31, 2016·International Journal of Molecular Sciences·Catherine StaudtMarielle Boonen
Jan 21, 2017·Current Opinion in Pediatrics·Koenraad R VeysElena Levtchenko
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May 1, 2020·Frontiers in Molecular Biosciences·Jacob M FavretDaesung Shin
Jan 12, 2021·Trends in Immunology·Mitchell Bijnen, Marc Bajénoff
May 13, 2021·Trends in Molecular Medicine·Amer JamalpoorManoe J Janssen
Jul 2, 2021·Frontiers in Immunology·Vaibhav TiwariDeepak Shukla

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