Myoblast transplantation was the first quasi-gene therapy to be suggested for Duchenne
muscular dystrophy. Animal experiments established the principles that the missing gene
could be targeted to muscle by grafting of genetically normal myoblasts that were able to
repair the disease-damaged muscle fibres. In the recipient muscle the gene was expressed
and the resultant protein provided some functional benefit in protecting the fibres against
necrosis. However, these effects were limited to a small region around the injection site and …