Exogenous Genes Are Expressed in mdx Muscle Fibres Following the Implantation of Primary Mouse Skin Cells

By implanting skin cells carrying exogenously introduced genes into the muscles of the x-linked muscular dystrophic (mdx) mouse, an animal model for human Duchenne muscular dystrophy, we show here that the introduced genes are expressed within the host muscle fibres. In one series of experiments the introduced gene was a lacZ reporter gene and in another the full length human dystrophin gene was introduced, via a germ line transgenic route, into the donor skin cells/The results indicate that skin cells could be used to carry genes to muscle fibres and further that the donor cells actually participate in the formation of muscle fibres in the host. Autologous skin cells of a myopathic patient might therefore be of use in designing therapies to alleviate myopathic conditions in cases where the disease is the result of a deficiency in a muscle specific protein. Key words: exogenous gene delivery, skin cells, muscle fibres. Basic Appl Myol. 7 (3&4): 211-219, 1997 In a previous paper we reported the formation of high numbers of newly-formed dystrophin-positive fibres whe