3 projets européens trouvés
Recherche sur 125080 projets européens
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
Biomarkers of renal graft injuries in kidney allograft recipients (BIOMARGIN)
Date du début: 1 mars 2013, Date de fin: 28 févr. 2018,
In renal allograft recipients, 10-year graft survival has not improved over the past decades. Histological examination of graft biopsies has long been the gold standard to confirm graft injuries, but biopsies are invasive and histological grading is not very robust. There is thus a need for robust, non-invasive methods to predict and diagnose acute and chronic graft lesions, to improve patient tre ...
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13
Participants partenaires
TERMINÉ
Phase I/II ex vivo gene therapy clinical trial for recessive dystrophic epidermolysis bullosa using skin equivalent grafts genetically corrected with a COL7A1-encoding SIN retroviral vector (GENEGRAFT)
Date du début: 1 mars 2011, Date de fin: 31 déc. 2017,
RDEB is one of the most severe rare genetic skin diseases of children and adults characterized by skin blistering resulting from lack of expression of type VII collagen. There is no treatment for this life-threatening disease. In March 2009, we obtained the orphan drug designation for the Medicinal product: “Skin equivalent graft genetically corrected with a COL7A1-encoding SIN retroviral vecto ...
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