Duchenne muscular dystrophy is a lethal condition caused by mutations in the dystrophin gene. Augmentation of the faulty copy by gene therapy represents a potential therapeutic answer, but delivery of functionally effective levels of the dystrophin gene in animal models has proved difficult. In the Early Edition of
So-called 'gutted' Ad vectors are modified such that all viral sequences are deleted, save those required for genome replication and packaging. DelloRusso et al. used vectors carrying either full-length human or mouse dystrophin cDNAs regulated by a powerful muscle-specific promoter. They observed that a single intramuscular injection restored dystrophin production to 25–30% of normal mouse limb muscle levels...