CORDIS Project
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This project aims to uncover the molecular mechanisms behind skeletal abnormalities in lysosomal storage disorders. It seeks to develop new therapeutic strategies to improve skeletal health by enhancing lysosomal function and promoting cellular clearance.
Lysosomal storage disorders (LSDs) are inherited diseases characterized by progressive intracellular accumulation of undigested macromolecules due to lysosomal dysfunction.
This results in a complex phenotype with broad pathological manifestations.
Most LSDs are characterized by defective skeletogenesis.
Despite this, the mechanisms by which lysosomal storage affects skeletal development and function is still unknown and the efficacy of current therapies on the skeletal system is limited.
This p…
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