Objective
Improvement of the utilization of the adenovirus as vector of transfer and expression of the gene of dystrophin in vivo: a potential treatment of Duchenne miopathy (DMD).
Among the viral vector candidates for human gene therapy in Duchenne muscular dystrophy, adenoviruses are particularly attractive. Adenoviruses have several interesting properties: they are weakly pathogenic in humans and they are easily amplified. Recent reports have demonstrated that the expression of foreign genes transferred by adenovirus vectors can be observed for long periods, especially in liver and skeletal muscles. The aim of the present study is the improvement of gene therapy of mdx mice, combining two strategies. The first is the construction of an adenovirus harboring the dystrophin cDNA under the control of a muscle specific promoter, making it possible to restrict the expression dystrophin only in muscular tissues. The second is to use systemic administration of recombinant adenovirus to express the dystrophin gene in all muscle (especially heart and diaphragm). The combination of these strategies can offer, in the near future, promising and encouraging results in the treatment of DMD patients.
Fields of science (EuroSciVoc)
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques. See: The European Science Vocabulary.
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques. See: The European Science Vocabulary.
- medical and health sciences medical biotechnology genetic engineering gene therapy
- medical and health sciences basic medicine neurology muscular dystrophies duchenne muscular dystrophy
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Funding scheme (or “Type of Action”) inside a programme with common features. It specifies: the scope of what is funded; the reimbursement rate; specific evaluation criteria to qualify for funding; and the use of simplified forms of costs like lump sums.
Coordinator
94805 Villejuif
France
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