FRRB Project 1745126 - A thorough understanding of the molecular players causing rare complement-mediated glomerular diseases toward a personalized therapy
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Name and Surname of PI |
Caterina Mele |
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Project Acronym |
UNEARTH |
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Project ID |
1745126 |
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Host Institution |
Istituto di Ricerche Farmacologiche Mario Negri IRCCS - Milan |
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Pathology of Interest |
C3 glomerulopathy / Immune-complex-mediated membranoproliferative glomerulonephritis |
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Research Area |
Rare Diseases / Nephrology |
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Project Start Date |
1 April 2021 |
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Project End Date |
31 March 2024 |
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Funding |
€ 526.000,00 |
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Type of Project |
Individual |
PROJECT SUMMARY
Immune-complex-mediated membranoproliferative glomerulonephritis (IC-MPGN) and C3 glomerulopathy (C3G) are rare kidney disorders, which lack an effective treatment. The prognosis is unfavourable and most patients develop end-stage renal failure during late childhood and early adolescence. Genetic defects leading to an uncontrolled activation of the complement system are found in C3G as frequently as in IC-MPGN. However, the genetic causes are still unknown in approximately 80% of patients, and the functional consequences of the genetic abnormalities have been investigated only in a few cases. Using a multidisciplinary approach, we will study the molecular, biochemical and cellular mechanisms through which the genetic alterations cause IC-MPGN/C3G. The results of these studies will be instrumental in characterising the pathogenetic mechanism underlying the disease in each patient, and will contribute to the development of personalised therapies.