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Understanding and predicting factor VIII tolerance – a key for precision medicine and cure in hemophilia A
| Funder | Swedish Research Council |
|---|---|
| Recipient Organization | Lund University |
| Country | Sweden |
| Start Date | Jan 01, 2023 |
| End Date | Dec 31, 2025 |
| Duration | 1,095 days |
| Number of Grantees | 3 |
| Roles | Principal Investigator; Co-Investigator |
| Data Source | Swedish Research Council |
| Grant ID | 2022-00841_VR |
Grant Description
Background:Hemophilia A (HA) is characterized by deficiency of factor VIII (FVIII).
A prerequisite for effective therapy, for future potential cure by gene therapy, and a minimized risk of complications will be an understanding and prediction of immunological tolerance towards exogenous FVIII.Purpose: To understand the mechanisms of FVIII tolerance in patients with severe HA and based on this knowledge develop a predictive model.Aims:1.
To develop a highly selective and sensitive assay for the identification of endogenous FVIII.2. To characterize genetic variants decisive for down-regulation of the immune response towards FVIII.3.
To develop a predictive model for FVIII tolerance Work plan: A selective and sensitive method for measuring FVIII levels
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Lund University
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