Gene therapy vectors, tissue-specific strategies

Another, relatively common, genetic disease that has the potential to be treated hy gene therapy is Duchenne muscular dystrophy (DMD). Most effort to replace the defective DMD gene has concerned an ex vivo strategy to transfect isolated muscle cells with multiple copies of the DMD gene and re-implant the cells back into the patient s muscle. There has so far been little work on the in vivo delivery of the DMD gene as there are few vectors suitable for the efficient delivery of genes into muscle cells in vivo and ensure tissue specific expression . 

---