7 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
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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10
Participants partenaires
TERMINÉ
Phase I/II Gene Therapy Trial of Fanconi anemia patients with a new Orphan Drug consisting of a lentiviral vector carrying the FANCA gene: A Coordinated International Action (EUROFANCOLEN)
Date du début: 1 janv. 2013, Date de fin: 31 déc. 2017,
Fanconi anemia (FA) is a rare inherited syndrome characterized by the early development of bone marrow failure and increasing predisposition to cancer with age. Allogeneic hematopoietic cell transplantation (alloHCT) is the only curative therapy for hematopoietic manifestations of FA, although associated with complications arising from myeloablation, graft versus host disease and increased inciden ...
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13
Participants partenaires
TERMINÉ
Gene Therapy for Inherited Skin Adhesion Disorders (GT-SKIN)
Date du début: 1 déc. 2011, Date de fin: 31 mai 2017,
Epidermolysis bullosa (EB) is a family of severe skin adhesion defects characterized by disfiguring blistering, infections, visual impairment, and a high risk of cancer. We showed through pre-clinical and clinical investigation that transplantation of genetically corrected epithelia leads to long-term functional correction of EB. The current vectors, however, integrate in an uncontrolled fashion i ...
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1
Participant partenaire
TERMINÉ
Cities demonstrating cybernetic mobility (CITYMOBIL2)
Date du début: 1 sept. 2012, Date de fin: 31 août 2016,
European cities face four main mobility problems: congestion, land use , safety and environment. One of the main causes of such problems is the car-ownership rate. The centres of large cities address this issue combining efficient mass transits with car restriction policies but peripheral areas and smaller cities remain dominated by private cars.CityMobil has demonstrated how automating road vehi ...
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49
Participants partenaires
TERMINÉ
Persisting Transgenesis (PERSIST)
Date du début: 1 janv. 2009, Date de fin: 30 juin 2013,
"For many disabling or fatal diseases, there is pre-clinical or clinical evidence of the potential therapeutic efficacy of gene therapy and, yet, the limitations of current gene transfer technologies have prevented success or even caused serious adverse events leading to termination of trials. PERSIST will explore the use of highly innovative gene-modifying and delivery technologies and capitalize ...
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29
Participants partenaires
TERMINÉ
Development of targeted DNA-Chips for High Throughput Diagnosis of NeuroMuscular Disorders (NMD-Chip)
Date du début: 1 oct. 2008, Date de fin: 30 sept. 2011,
Inherited Neuromuscular Diseases (NMD) form a large group of diseases including Limb-Girdle Muscular Dystrophies (LGMD), Congenital Muscular Dystrophies (CMD), Duchenne and Becker Muscular Dystrophies (DMD/BMD), or Charcot-Marie-Tooth disease (CMT). Within a given disease group genetic and clinical heterogeneity is the hallmark. The precise diagnostic of neuromuscular diseases thus requires extens ...
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