Förståelse och prediktion av faktor VIII tolerans - en nyckel till precisionsmedicin och bot vid hemofili A

Projektinformation

Kort sammanfattning

To understand the biochemical mechanisms underlying how immune tolerance towards the deficient factor VIII (FVIII) provided by replacement therapy occurs in patients with severe hemophilia A (HA) despite having no measurable endogenous (self) protein. Based on this knowledge, to develop a predictive tolerance model to facilitate precision medicine for optimal care in an era of several new therapeutic options including gene therapy.

Beskrivning

Detta är ett Vetenskapsråds finansierat projekt under åren 2023-2026.
1. To develop a novel highly selective and sensitive analytical method for the detection of endogenous FVIII protein (self) in patients with no measurable FVIII antigen using standard assays (WP1)
2. To identify and characterize genetic variants modifying the immune response towards exogenous FVIII (non-self) (WP2)
3. To develop a predictive model for FVIII tolerance in severe HA (WP3)
StatusPågående
Gällande start-/slutdatum23-01-01 → …