mmunosuppressant drugs, such as FK506may be necessary to fully overcome the immune response to adenovirus-based genetherapy. All current gene based research has been performed on animals, but this fall,investigators at the University of Ohio-Columbus and the University of Michigan-AnnArbor will begin a very limited human trial of gene therapy in Duchenne's musculardystrophy. The major goal of the 24 week study is to establish the safety of the genetransfer procedure. The study involves 12 participants with Duchenne's musculardystrophy and is waiting for final approval from the Food and Drug Administration. Another focus of research on Duchenne's muscular dystrophy involves the proteinUtrophin. Utrophin is almost exactly like dystrophin, and its potential as a replacement fordystrophin has stirred much interest. Utrophin genes could be introduced into the bodyvia an adenovirus (described above) and "fill in" for the missing dystrophin protein. Themajor advantage of utrophin over dystrophin is that individuals with the disorder alreadymake utrophin, so their immune systems would accept the protein and not reject it asforeign. Utrophin is coded for on chromosome 6 and is thus unaffected by the defective Xchromosome. Therefore, another method of increasing utrophin would be to manipulatethe utrophin genes already present in the muscle fibers to produce more. Utrophin isnormally found only at the neuromuscular junction, but to be effective, it must completelysurround the muscle fiber. Researchers have found that during fetal life, humans exhibitutrophin around the entire muscle fiber, but as development progresses, the utrophin isreplaced with dystrophin. Investigators hope to find the "switch" that creates this changeand reverse its effects....
