CORDIS Project
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This project aims to improve gene therapy for Wiskott-Aldrich Syndrome by using lentiviral vectors to restore immune function in affected patients. It focuses on enhancing the safety and efficiency of gene transfer methods in hematopoietic stem cells.
Wiskott-Aldrich Syndrome (WAS) is a primary X-linked rare disease (1:250.000 mainly children) caused by mutations in the was gene.
Gene Therapy (GT) results in Primary Immunodeficiencies (PID) have been particularly successful due to advances in gene modification of hematopoietic stem cells (HSCs).
Currently there is a clinical trial which has developed an ex vivo approach that uses a HIV-derived lentiviral vector to transfer genes into autologous CD34+ HSCs from WAS patients.
This clinical stud…
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