Lecture 'Gene therapy of primary immunodeficiencies. What have we learned so far?'

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Students, Employees, Alumni, Business
27-03-2019 from 11:30 to 12:45
UGent-VIB-onderzoeksgebouw, Technologiepark 71, 9052 Zwijnaarde
VIB-UGent Center for Inflammation Research (GE01/WE14/WE10)
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Prof. Alain Fischer, Collège de France, AP-HP, Imagine Institute Paris - PID, Gene therapy

The first success of gene therapy is now 20 years old. It consisted in the treatment of SCID X1 (common Gamma chain deficiency) by ex vivo gene transfer into hematopoietic progenitor cells by using a retroviral vector

The first generation of these vectors turned out to be causing insertional mutagenesis in some cases. Thus a second generation was developed that, together withe usage of more potent lentiviral vectors has enabled to treat worldwide, safely and with sustained efficacy more than 200 patients affected by 3 forms of immunodeficiencies. These results open the perspective to treat many more of these genetic disorders of the immune system.

Finally, surrogate gene marking of lymphocyte populations from treated patients offers opportunities to study the dynamics of these cells in vivo