title: Gene Therapy for Duchenne muscular dystrophy.
abstract: BACKGROUND
  Duchenne muscular dystrophy (DMD) is a relatively common inherited disorder caused by defective expression of the protein dystrophin. The most direct approach to treating this disease would be to restore dystrophin production in muscle. Recent progress has greatly increased the prospects for successful gene therapy of DMD, and here we summarize the most promising developments.
  
  
  METHODS
  Gene transfer using vectors derived from adeno-associated virus (AAV) has emerged as a promising method to restore dystrophin production in muscles bodywide, and represents a treatment option applicable to all DMD patients. Using information gleaned from PubMed searches of the literature, attendance at scientific conferences and results from our own lab, we provide an overview of the potential for gene therapy of DMD using AAV vectors including a summary of promising developments and issues that need to be resolved prior to large-scale therapeutic implementation.
  
  
  CONCLUSIONS
  Of the many approaches being pursued to treat DMD and BMD, gene therapy based on AAV-mediated delivery of microdystrophin is the most direct and promising method to treat the cause of the disorder. The major challenges to this approach are ensuring that microdystrophin can be delivered safely and efficiently without eliciting an immune response.
authors.affiliation: University of Washington, Wellstone Muscular Dystrophy Research Center, Department of Neurology, Seattle, WA, 98195-7720, USA.
authors.forename: Julian
authors.initials: J
authors.lastname: Ramos
authors.affiliation: University of Washington, Wellstone Muscular Dystrophy Research Center, Department of Neurology, Seattle, WA, 98195-7720, USA.
authors.forename: Jeffrey S
authors.initials: JS
authors.lastname: Chamberlain
keywords: Becker muscular dystrophy (BMD); Duchenne muscular dystrophy (DMD); Dystrophin; adeno-associated viral (AAV) vectors; gene therapy; immune response; micro-dystrophin; micro-utrophin; tolerization