CORDIS Project
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This project focuses on developing gene therapy techniques using modified stem cells to restore immune systems in patients with severe combined immunodeficiency and other genetic disorders. It also aims to enhance stem cell transplantation methods to improve immune recovery in affected individuals.
We have developed the tools and experimental models and have provided the proof of principle that gene modified autologous haematopoietic stem cells can restore a complete immunological system in vivo in a particular form of SCID.
We are pursuing these efforts applying this strategy to several other diseases : two lentivirus protocols for X-linked adrenoleukodystrophy, Thalassemia and sickle cell disease, are developed in our Clinical Investigation Centre.
In 2010, we plan to initiate two other…
INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE
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