MUSCLE-BASED GENE-THERAPY - REALISTIC POSSIBILITIES FOR THE FUTURE

The past five years have witnessed tremendous growth in the field of g ene therapy, with pre-clinical and clinical gene therapy trials for di seases as diverse as cancer, AIDS and atherosclerosis, These studies h ave utilized many different vectors and target organs in order to achi eve therapeutic effects, In this review, we examine the rationale for using skeletal muscle as a target tissue for gene therapy, discuss the wide array of vectors that have been used for muscle-based gene thera py, summarize the disease-targets that have been approached using thes e techniques, and discuss some of the obstacles that remain to be over come en route to successful muscle-based human gene therapy.