There are a few ideas about which cells could be used. Johnny Huard at the University of Pittsburgh described his moderately successful experiments using it as a gene therapy vector in mice, after altering it to carry a new gene. The trials failed to show any functional effect, and few of the cells survived, probably because of the body's immune response to the donated cells and characteristics of the cells themselves. The myoblast is relatively young and flexible, but some experts say it's still too mature, too far along in its development, to be an ideal gene therapy vector. Jennifer Morgan of Hammersmith Hospital in London said her group may have identified such a cell.
Skin cells are far easier to extract than muscle cells, so the idea is intriguing.
Immune-system cells known as macrophages and monocytes could also be used to the gene therapist's advantage, said George Dickson. Why not make them the bearers of a new gene, Dickson suggested. They're easy to extract, since they circulate in the blood, and they're easy to grow in the lab, where they can be made to carry a therapeutic gene. The only part that seems safe to condense is the rod section. Some people with very mild muscular dystrophy have parts of this section missing.