• Title of article

    Treatment of lysosomal storage disorders: Focus on the neuronal ceroid-lipofuscinoses

  • Author/Authors

    Chris Pierret، نويسنده , , Jason A. Morrison، نويسنده , , Mark D. Kirk، نويسنده ,

  • Issue Information
    روزنامه با شماره پیاپی سال 2008
  • Pages
    14
  • From page
    429
  • To page
    442
  • Abstract
    Recent advances in our understanding of lysosomal storage disorders (LSDs) may lead to new therapies to treat the neuronal ceroid-lipofuscinoses (NCLs). In this review, enzyme replacement therapy, gene therapy, cell-mediated therapy and pharma ceutical treatments are considered across the LSDs and extended to therapies for the NCLs. It is likely that a combination of approaches will produce the most beneficial clinical outcome for treatment of pathologies displayed by the NCLs.
  • Keywords
    neuronal ceroid-lipofuscinoses , lysosomal storage , enzyme replacement , Stem cell , gene therapy , substrate reduction
  • Journal title
    Acta Neurobiologiae Experimentalis
  • Serial Year
    2008
  • Journal title
    Acta Neurobiologiae Experimentalis
  • Record number

    672743