Effect of Clodronate on the Pathogenesis in a Mouse Model of Lysosomal Acid Lipase Deficiency.
4 affiliations
- 1Department of Clinical Laboratory Medicine, National Center for Child Health and Development, 2-10-1 Okura, Setagaya-ku, Tokyo 157-8535, Japan.
- 2Department of Systems BioMedicine, National Research Institute for Child Health and Development, 2-10-1 Okura, Setagaya-ku, Tokyo 157-8535, Japan.
- 3Diagnostics and Therapeutics of Intractable Diseases, Intractable Disease Research Center, Graduate School of Medicine, Juntendo University, Bunkyo-ku, Tokyo 113-8421, Japan.
- 4Department of Pediatrics, Graduate School of Medicine, Osaka Metropolitan University, Osaka 545-8585, Japan.
Abstract
Lysosomal acid lipase deficiency (LAL-D, OMIM #309900), caused by mutations of the LIPA gene, is involved in two continuum disease phenotypes: Wolman disease in infants and cholesteryl ester storage disease in adults. Their major phenotypes include steatosis, hepatomegaly, splenomegaly, and elevated liver enzymes. Enzyme replacement therapy has been used for the treatment of LAL-D. In this study, we examined the therapeutic effect of liposomal clodronate in an LAL-D mouse model. When liposomal clodronate was administered intraperitoneally by a single administration at 200 mg/kg to adult mice, organ cholesterol and triglyceride levels partially decreased. Furthermore, a significant correction of the relative organ weight of the liver and spleen as well as a reduction in splenic lipids were observed when liposomal clodronate was administered at 50 mg/kg to young mice intraperitoneally by multiple administrations. These results demonstrate that liposomal clodronate might have a therapeutic effect on the LAL-D mouse model.
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BibTeX
@article{mashima2026effect,
title = {{Effect of Clodronate on the Pathogenesis in a Mouse Model of Lysosomal Acid Lipase Deficiency.}},
author = {Ryuichi Mashima and Shuji Takada and Kei Murayama and Takashi Hamazaki},
journal = {International journal of molecular sciences},
year = {2026},
month = sep,
doi = {10.3390/ijms27198694},
url = {https://europepmc.org/article/MED/42883938},
note = {CC BY},
}
RIS
TY - JOUR
TI - Effect of Clodronate on the Pathogenesis in a Mouse Model of Lysosomal Acid Lipase Deficiency.
AU - Ryuichi Mashima
AU - Shuji Takada
AU - Kei Murayama
AU - Takashi Hamazaki
PY - 2026
DA - 2026/09/29
JO - International journal of molecular sciences
DO - 10.3390/ijms27198694
UR - https://europepmc.org/article/MED/42883938
AB - Lysosomal acid lipase deficiency (LAL-D, OMIM #309900), caused by mutations of the LIPA gene, is involved in two continuum disease phenotypes: Wolman disease in infants and cholesteryl ester storage disease in adults. Their major phenotypes include steatosis, hepatomegaly, splenomegaly, and elevated liver enzymes. Enzyme replacement therapy has been used for the treatment of LAL-D. In this study, we examined the therapeutic effect of liposomal clodronate in an LAL-D mouse model. When liposomal clodronate was administered intraperitoneally by a single administration at 200 mg/kg to adult mice, organ cholesterol and triglyceride levels partially decreased. Furthermore, a significant correction of the relative organ weight of the liver and spleen as well as a reduction in splenic lipids were observed when liposomal clodronate was administered at 50 mg/kg to young mice intraperitoneally by multiple administrations. These results demonstrate that liposomal clodronate might have a therapeutic effect on the LAL-D mouse model.
SN - 1422-0067
ER -