So the objective of gene therapy in these disorders is to correct the defect that causes the essential protein dystrophin to be absent or altered in affected individuals. In ADA deficiency, the correction is made in cells that can be taken from the bloodstream and then reintroduced into the bloodstream. In CF the targets for correction are cells in the lungs. DMD is much more difficult. The correction must be made in the cells of the skeletal muscle which are not so easily accessible.
Myoblast transfer is a localized procedure requiring many injections.
It can be considered a form of gene therapy in that the muscle cell is the vehicle carrying the corrected dystrophin gene to the muscle where it is needed. The correct message triggers the production of normal dystrophin. Ideally, gene therapy in DMD would be accomplished through the bloodstream and will compensate for missing dystrophin in all affected muscle groups throughout the body. A few years ago we heard promising things about myoblast transfer. I would like to request contact information for researchers or doctors who will, privately, talk candidly.