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Project
Immune stealth nanotechnology for gene therapy of hemophilia 'A': mechanisms and translational implications (FWOAL651)
Hemophilia B is a genetic disease due to a defective gene that encodes clotting factor IX (FIX). Current treatment requires injection of purified FIX protein. Though this has improved patients' quality of life it is not a cure and serious bleeding still occurs.Moreover, the patients' immune system can recognize the FIX protein as "foreign".
This results in the induction of antibodies, called "inhibitors", that inactivate FIX and increase the risk of life-threatening bleeds. Hence, there is a need to develop new therapies for hemophilia that allow for sustained FIX production and minimize the risk of developing inhibitors.
This could be achieved by introducing a functional FIX gene into the patient's cells using gene therapy. The objective of the present project proposal is to develop a more efficient and safer gene therapy approach using "immune stealth" nanoparticles containing the FIX gene. These "immune stealth" nanoparticles are specifically designed to make the genetically modified cells that produce FIX after gene therapy invisible to the immune system. If successful, this approach may ultimately lead to a new strategy that may lead to a cure of hemophilia B and prevent "inhibitor" development with broad implications for gene therapy of other diseases.
This results in the induction of antibodies, called "inhibitors", that inactivate FIX and increase the risk of life-threatening bleeds. Hence, there is a need to develop new therapies for hemophilia that allow for sustained FIX production and minimize the risk of developing inhibitors.
This could be achieved by introducing a functional FIX gene into the patient's cells using gene therapy. The objective of the present project proposal is to develop a more efficient and safer gene therapy approach using "immune stealth" nanoparticles containing the FIX gene. These "immune stealth" nanoparticles are specifically designed to make the genetically modified cells that produce FIX after gene therapy invisible to the immune system. If successful, this approach may ultimately lead to a new strategy that may lead to a cure of hemophilia B and prevent "inhibitor" development with broad implications for gene therapy of other diseases.
Date:1 Jan 2012 → 31 Dec 2015
Keywords:Fibrosis, Hepatic Stellate Cells, Histon (de)acetylation, Stellate cell activation, Liver Cell Transplantation, Liver Sinusoidal Cells, Sinusoidal Cells, Portal hypertension, cirrhosis, Cytoskeleton, Cell Biology, Fat-Storing Cells, NASH / NAFLD, Intermediate Filaments, liver stem / progenitor cells, Flow Cytometry, Metabolic Syndrome
Disciplines:Electrical and electronic engineering, Basic sciences, Biological sciences