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Int. J. Mol. Sci. 2018, 19(11), 3599;

Gadolinium Chloride Rescues Niemann–Pick Type C Liver Damage

Centro de Genética y Genómica, Facultad de Medicina, Clínica Alemana Universidad del Desarrollo, Santiago 7590943, Chile
Departamento de Gastroenterología, Facultad de Medicina, Pontificia Universidad Católica de Chile, Santiago 8331150, Chile
Center for Genomic and Bioinformatic, Faculty of Sciences, Universidad Mayor, Santiago 8580745, Chile
Authors to whom correspondence should be addressed.
Received: 29 September 2018 / Revised: 1 November 2018 / Accepted: 2 November 2018 / Published: 14 November 2018
(This article belongs to the Special Issue Molecular Features of Lysosomal Storage Disorders)
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Niemann–Pick type C (NPC) disease is a rare neurovisceral cholesterol storage disorder that arises from loss of function mutations in the NPC1 or NPC2 genes. Soon after birth, some patients present with an aggressive hepatosplenomegaly and cholestatic signs. Histopathologically, the liver presents with large numbers of foam cells; however, their role in disease pathogenesis has not been explored in depth. Here, we studied the consequences of gadolinium chloride (GdCl3) treatment, a well-known Kupffer/foam cell inhibitor, at late stages of NPC liver disease and compared it with NPC1 genetic rescue in hepatocytes in vivo. GdCl3 treatment successfully blocked the endocytic capacity of hepatic Kupffer/foam measured by India ink endocytosis, decreased the levels CD68—A marker of Kupffer cells in the liver—and normalized the transaminase levels in serum of NPC mice to a similar extent to those obtained by genetic Npc1 rescue of liver cells. Gadolinium salts are widely used as magnetic resonance imaging (MRI) contrasts. This study opens the possibility of targeting foam cells with gadolinium or by other means for improving NPC liver disease. Synopsis: Gadolinium chloride can effectively rescue some parameters of liver dysfunction in NPC mice and its potential use in patients should be carefully evaluated. View Full-Text
Keywords: Niemann–Pick type C disease; gadolinium chloride; liver disease; cholesterol; therapeutic option Niemann–Pick type C disease; gadolinium chloride; liver disease; cholesterol; therapeutic option

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This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited (CC BY 4.0).

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Klein, A.D.; Oyarzún, J.E.; Cortez, C.; Zanlungo, S. Gadolinium Chloride Rescues Niemann–Pick Type C Liver Damage. Int. J. Mol. Sci. 2018, 19, 3599.

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