ABSTRACT
This chapter reviews the development of gene transfer technology using muscles as a drug delivery vehicle. Specifically, the myoblast-mediated gene transfer method is discussed in detail for its advantages, potential problems, applications, and future prospects in human gene therapy. Myoblasts in culture can be modified genetically and reimplanted into muscle tissue, where they fuse with the host myofibers or with themselves, generating new recombinant myofiber cells, which can continue producing the recombinant product. Myoblast-mediated gene transfer was originally tested with C2C12 cells, an established myoblast cell line, for its feasibility in producing growth hormone genes, factor IX gene, and ß-galactosidase. An interesting paradigm of the myoblast-mediated gene transfer approach is to achieve ectopic expression of foreign genes by explanting genetically modified myoblasts in tissues other than muscles. As a general method that assures a stable gene transfer and long-term production of transgenes, the myoblast-mediated gene transfer method has been tested extensively for its feasibility.
