Improvement of the utilization of the adenovirus as vector of transfer and expression of the gene of dystrophin in vivo a potential traetement of duchenne miophaty

Линиите свързват координатора с партньорите. За проекти отпреди 2014 г. CORDIS не винаги дава точни координати. Тези точки са на ниво град или държава.

Накратко на български

Цел на проекта

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.

Оригинален текст от CORDIS (на английски).

Участници

• Centre National de la Recherche Scientifique (CNRS) · PARISКоординаторФранция
• Not availableНиво градИспания

Връзки

• Виж в CORDIS