CORDIS Project
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The RE-STORE project aims to develop a novel platform for screening potential therapies targeting skeletal issues in lysosomal storage disorders. By focusing on enhancing the degradation of endoplasmic reticulum fragments, the project seeks to identify new treatments for patients suffering from these genetic diseases.
Lysosomal storage disorders (LSDs) are a family of inherited genetic diseases characterized by lysosomal dysfunction, with a consequent block in the degradative capacity of the cell.
The skeleton is one of the most affected organs in LSD patients and current therapies are largely ineffective for the treatment of bone and cartilage manifestations.
Thanks to the support of the ERC-starting grant BONEPHAGY we demonstrated that the degradation of the endoplasmic reticulum fragments via lysosome/aut…
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Italy, Pomezia Rm
Type: SME
Activity type: Private for-profit entities (excluding Higher or Secondary Education Establishments)
SME: Yes
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