Muscling in: Gene therapies for muscular dystrophy target RNA.
Muscle diseases can take many forms, from the progressive muscle degeneration of dystrophies to the childhood cancer rhabdomyosarcoma. In 'Bench to Bedside', Joel R. Chamberlain and Jeffrey S. Chamberlain discuss studies using antisense oligonucleotides to treat Duchenne muscular dystrophy and myotonic dystrophy. In 'Bedside to Bench', Simone Hettmer and Amy J. Wagers examine the implications of clinical studies describing a type of rhabdomyosarcoma that resembles acute leukemia. The findings dovetail with other studies suggesting that some of these cancers might originate outside of muscle tissue and highlight the need for a better understanding of the cells that give rise to this condition.
Division of Medical Genetics, Department of Medicine, University of Washington, Seattle, Washington, USA. jrcham@uw.edu
Joel R
JR
Chamberlain
Jeffrey S
JS
Chamberlain
D000818:Animals; D004195:Disease Models, Animal; D015316:Genetic Therapy; D051379:Mice; D009136:Muscular Dystrophies / Q000235:genetics*; D016376:Oligonucleotides, Antisense / Q000235:genetics; D012313:RNA / Q000235:genetics*
D016376:Oligonucleotides, Antisense; D012313:RNA