2 projets européens trouvés
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TERMINÉ
Gene Therapy for X-linked Chronic Granulomatous Disease (CGD) (NET4CGD)
Date du début: 1 déc. 2012, Date de fin: 31 mai 2018,
This project is focused on the clinical development of a new orphan drug that can rapidly become a new treatment option for patients with the X-linked form of chronic granulomatous disease (X-CGD). This rare primary immune deficiency of phagocytes is caused by mutations in the gp91phox gene. Affected patients are highly-susceptible to infections and develop inflammatory granulomas. Several member ...
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11
Participants partenaires
TERMINÉ
Advanced Cell-based Therapies for the treatment of Primary ImmunoDeficiency (CELL-PID)
Date du début: 1 nov. 2010, Date de fin: 30 avr. 2016,
Primary immune deficiencies (PID) are inherited disorders of the adaptive and innate immune system marked by severe infections, autoimmunity and high risk of cancer. Treatment entails hematopoietic stem cell (HSC) transplantation from allogeneic donors, however in the absence of an HLA compatible donor, HSCT outcome is limited by delayed or suboptimal reconstitution and complications. SCID-X1 and ...
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