Susan Brown/Jack Lucy: Dystrophin. Gene, protein, and cell biology. Cambridge UP 1997. 0-521-55033-5. $90. 13754 Stephen Tapscott: Deconstructing myotonic dystrophy. Science 8 September 2000, 1701-1702. 5589 Jeremy Webb: Mini-gene injection prevents muscle-wasting disease. New Scientist 28 August 1993, 8-9. Gene therapy has cured mice of a muscle-wasting disorder similar to Duchenne muscular dystrophy, a fatal disease that kills boys in their teens or early twenties. It is one of the most common and severe disorders to be cused by a single defective gene, it is inherited on the X chromosome, and the responsible gene is the biggest known human gene (with 2 MBP). Sufferers fail to make dystrophin, a protein essential for maintaining the structure and strength of muscle cells.