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12 projets européens trouvés

Recherche sur 125080 projets européens

 TERMINÉ 

Muscle Stress Relief: An integrated research program linking together basic research on secondary myopathies in stress states to innovative translation in applied myology. (Muscle stress relief)

Date du début: 1 mars 2016, Date de fin: 29 févr. 2020,

A diverse variety of medical or lifestyle conditions lead to a progressive loss of muscle force by functionally impairing myofibril contractility and causing ultimately myofibril loss. Major underlying risk factors of chronic muscle force loss are ageing, inactive lifestyles, and unbalanced nutrition. Together, these factors are predicted to lead to an endemic incidence of muscle weakness both in ...
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 14

 TERMINÉ 

DevelopIng Genetic medicines for Severe Combined Immunodeficiency (SCID) (SCIDNET)

Date du début: 1 janv. 2016, Date de fin: 31 déc. 2019,

Severe combined immunodeficiency (SCID) is a devastating rare disorder of immune system development. Affected infants are born without functional immune systems and die within the first year of life unless effective treatment is given. Treatment options are limited to allogeneic haematopoietic stem cell transplantation and autologous stem cell gene therapy. Over the last 15 years, gene therapy for ...
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 13

 TERMINÉ 

Development of an innovative gene therapy platform to cure rare hereditary muscle disorders (MYOCURE)

Date du début: 1 janv. 2016, Date de fin: 31 déc. 2019,

The objective of MYOCURE is to develop an innovative gene therapy platform to cure rare hereditary muscle disorders, specifically focusing on myotubular myopathy (MTM) and glycogen storage disorder (GSD) type II. These are attractive diseases for gene therapy since they compromise a diverse family of rare genetic diseases typically caused by single gene defects that often provoke significant morbi ...
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 8

 TERMINÉ 

Efficacy and safety of low-dose IL-2 (ld-IL-2) as a Treg enhancer for anti-neuroinflammatory therapy in newly diagnosed Amyotrophic Lateral Sclerosis (ALS) patients (MIROCALS)

Date du début: 1 sept. 2015, Date de fin: 31 août 2019,

Amyotrophic Lateral Sclerosis (ALS) is a fatal degenerative disorder of the brain and spinal cord affecting some 40,000 individuals in Europe, causing 11,000 deaths each year. Our pioneering work on riluzole showed that it is possible to modify ALS progression but all subsequent trials of potential neuroprotective agents have failed. Thus, drug development in ALS, including trial design, patient s ...
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 12

 TERMINÉ 

Correction of duplications in the DMD gene by a CRISPR/Cas9 approach (DMD2CURE)

Date du début: 9 janv. 2017, Date de fin: 8 janv. 2019,

Duchenne muscular dystrophy is an X-linked recessive muscle-wasting disease, characterized by progressive weakening of skeletal, respiratory, and cardiac muscle followed by necrosis and fibrosis. DMD affects ~1:3,500 live male births and is associated with delayed motor milestones. DMD occurs as a result of mutations within the DMD gene that lead to premature termination of translation. The most f ...
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 1

 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

 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

 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

 TERMINÉ 

Genetic correction of glycogen debranching enzyme deficiency in Glycogen Storage disease III: a proof of concept study (GLYCODIS3)

Date du début: 1 janv. 2016, Date de fin: 31 déc. 2017,

Glycogen storage disease III (GSD III) is a rare (1:100,000) autosomal recessive disorder that results from the deficiency of the glycogen debranching enzyme (GDE). The major cause of morbidity is associated with the muscle accumulation of glycogen, which leads to progressive myopathy. A dietary treatment with frequent meals high in carbohydrates, slows the progression of the pathology that is how ...
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 1

 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

 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

 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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 13