title: MicroRNA and mRNA Expression Changes in Steroid Naïve and Steroid Treated DMD Patients.
abstract: BACKGROUND
  Duchenne Muscular Dystrophy (DMD) is a recessive X-linked form of muscular dystrophy. Steroid therapy has clinical benefits for DMD patients, but the mechanism remains unclear.
  
  
  OBJECTIVE
  This study was designed to identify mRNAs and microRNAs regulated in Duchenne Muscular Dystrophy patients prior to and after steroid therapy.
  
  
  METHODS
  Genome wide transcriptome profiling of whole blood was performed to identify mRNAs and microRNAs regulated in DMD patients.
  
  
  RESULTS
  The data show many regulated mRNAs and some microRNAs, including some muscle-specific microRNAs (e.g., miR-206), that were significantly altered in blood of young (age 3-10) DMD patients compared to young controls. A total of 95 microRNAs, but no mRNAs, were differentially expressed in older DMD patients compared to matched controls (age 11-20). Steroid treatment reversed expression patterns of several microRNAs (miR-206, miR-181a, miR-4538, miR-4539, miR-606, and miR-454) that were altered in the young DMD patients. As an example, the over-expression of miR-206 in young DMD patients is predicted to down-regulate a set of target genes (e.g., RHGAP31, KHSRP, CORO1B, PTBP1, C7orf58, DLG4, and KLF4) that would worsen motor function. Since steroids decreased miR-206 expression to control levels, this could provide one mechanism by which steroids improve motor function.
  
  
  CONCLUSIONS
  These identified microRNA-mRNA alterations will help better understand the pathophysiology of DMD and the response to steroid treatment.
authors.affiliation: Department of Neurology and the M.I.N.D. Institute, University of California at Davis, Sacramento, California, USA.
authors.forename: Da Zhi
authors.initials: DZ
authors.lastname: Liu
authors.affiliation: Department of Neurology and the M.I.N.D. Institute, University of California at Davis, Sacramento, California, USA.
authors.forename: Boryana
authors.initials: B
authors.lastname: Stamova
authors.affiliation: Division of Pediatric Neurology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
authors.forename: Shengyong
authors.initials: S
authors.lastname: Hu
authors.affiliation: Department of Neurology and the M.I.N.D. Institute, University of California at Davis, Sacramento, California, USA.
authors.forename: Bradley P
authors.initials: BP
authors.lastname: Ander
authors.affiliation: Department of Neurology and the M.I.N.D. Institute, University of California at Davis, Sacramento, California, USA.
authors.forename: Glen C
authors.initials: GC
authors.lastname: Jickling
authors.affiliation: Department of Neurology and the M.I.N.D. Institute, University of California at Davis, Sacramento, California, USA.
authors.forename: Xinhua
authors.initials: X
authors.lastname: Zhan
authors.affiliation: Department of Neurology and the M.I.N.D. Institute, University of California at Davis, Sacramento, California, USA.
authors.forename: Frank R
authors.initials: FR
authors.lastname: Sharp
authors.affiliation: Division of Pediatric Neurology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
authors.forename: Brenda
authors.initials: B
authors.lastname: Wong
keywords: Duchenne muscular dystrophy (DMD); blood; mRNA (gene); microRNA (miRNA)