Development of novel "immune stealth" nanotechnology for gene therapy of hemophilia: pre-clinical validation and underlying mechanisms

Project Details


Hemophilia is a severe bleeding disorder that is due to a defect in a gene encoding an essential clotting factor. Patients require life-long injections of purified clotting factors. This improves the patients' quality of life, but unfortunately patients can still suffer from recurrent, sometimes life-threatening bleeds. Sometimes the patients' immune system neutralizes the clotting factors, making treatment essentially ineffective. To overcome these limitations, we wanted to explore the possibility of using gene therapy instead.
Effective start/end date1/10/1330/06/14


  • Fibrosis
  • Hepatic Stellate Cells
  • Histon (de)acetylation
  • Stellate cell activation
  • Liver Cell Transplantation
  • Liver Sinusoidal Cells
  • Portal hypertension
  • Sinusoidal Cells
  • cirrhosis
  • Cytoskeleton
  • Cell Biology
  • Fat-Storing Cells
  • Intermediate Filaments
  • liver stem / progenitor cells
  • autophagy
  • Flow Cytometry
  • Metabolic Syndrome

Flemish discipline codes in use since 2023

  • Biological sciences
  • Electrical and electronic engineering
  • Basic sciences


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