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pro vyhledávání: '"Victoria V. Skopenkova"'
Autor:
Anna V. Starikova, Victoria V. Skopenkova, Anna V. Polikarpova, Denis A. Reshetov, Svetlana G. Vassilieva, Oleg A. Velyaev, Anna A. Shmidt, Irina M. Savchenko, Vladislav O. Soldatov, Tatiana V. Egorova, Maryana V. Bardina
Publikováno v:
Scientific Reports, Vol 12, Iss 1, Pp 1-13 (2022)
Abstract High expectations have been set on gene therapy with an AAV-delivered shortened version of dystrophin (µDys) for Duchenne muscular dystrophy (DMD), with several drug candidates currently undergoing clinical trials. Safety concerns with this
Externí odkaz:
https://doaj.org/article/e55381cec5004eccbc9982cbd02611bd
Publikováno v:
Acta Naturae
Many genetic diseases that are responsible for muscular disorders have been described to date. Gene replacement therapy is a state-of-the-art strategy used to treat such diseases. In this approach, the functional copy of a gene is delivered to the af