RGM.20.001 – CURE4LIFE: Developing stem cell based genetic medicines

Route: Regenerative medicine: game changer moving to broad areas of application

Cluster question: 096 How can we improve diagnostics, treatment, and vaccines for immunodeficiencies and infectious diseases?

In regenerative medicine, scientists and doctors work together to restore tissues that have been damaged by disease. Stem cells, capable of regenerating damaged tissues, are often used. However, in genetic diseases, stem cells of the patient carry disease-causing mutations. A way around this is to repair the harmful genes in stem cells and transplant these back into the patient. This is called gene therapy, which we will develop for five lethal diseases that can be cured with healthy blood stem cells. For all five diseases, there is an unmet medical need. Their current treatments are cumbersome, non-curative, fail to treat symptoms in the brain, or need to be given lifelong via injections, which can cause infections. The proposed gene therapy approach is curative and constitutes a one-time treatment, which will reduce the overall costs significantly. As most of these applications are currently done in children, the results would help establish a critical knowledge base for stem cell based gene therapy in adults. Scientifically, new information will be gathered on how the body restores the blood and immune system. Moreover, the platform we develop can be used for many other diseases that can be treated by the same principles.

Keywords

gene therapy, lymphocytes, metabolic diseases, red cells

Other organisations

AMC, EMC, Sanquin, SAS, Umijmegen, Utrecht

Submitter

Organisation Leids Universitair Medisch Centrum (LUMC)
Name Prof.dr. F. (Frank) Staal
E-mail f.j.t.staal@lumc.nl
Website https://immunology.lumc.nl/about-the-frank-staal-group-47