Muscle-mediated gene therapy.

: Transfer of therapeutic genes into muscle tissue holds promise for the treatment of a variety of muscular dystrophies, serum protein deficiencies and vascular proliferative disorders. Recent progress achieved in development of improved vectors allowed prolonged and efficient expression of genes encoding therapeutic proteins in muscle cells. The most important advances include: novel plasmid DNA vectors and methods for their efficient transfection in vivo, helper-dependent adenoviral vectors, allowing long-term gene expression and effective readministration in immunocompetent hosts and adeno-associated vectors. On the other hand, recent progress in this field has been facilitated by development of systems that enable regulated therapeutic gene expression in muscle tissue.