DIRECT GENE-TRANSFER INTO MUSCLE

Gene therapy has great promise for the treatment and the prevention of a broad range of inherited and acquired diseases. Gene transfer metho ds currently explored include the use of viral vectors and physical-ch emical methods. Plasmid DNA can be taken up by skeletal muscle cells i n vivo without any special delivery mechanism and persist long-term in an extrachromosomal, non-replicative circular form. Thus, foreign gen es can be expressed permanently in skeletal muscle. Ar present the eff iciency of gene transfer is not high enough to treat genetic muscle di seases. However, even at the relatively low efficiency of expression w e are able to achieve at present, plasmid DNA transfer seems to be a v ery promising way of programming cells in vivo to secrete proteins for immunization purposes.