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

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

Research output: Contribution to journalReview articlepeer-review

18 Scopus citations


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 pharmaceutical 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.

Original languageEnglish (US)
Pages (from-to)429-442
Number of pages14
JournalActa Neurobiologiae Experimentalis
Issue number3
StatePublished - 2008


  • Enzyme replacement
  • Gene therapy
  • Lysosomal storage
  • Neuronal ceroid-lipofuscinoses
  • Stem cell
  • Substrate reduction

ASJC Scopus subject areas

  • Neuroscience(all)


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