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

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.

• Publication year

  2008

• Venue

  Acta Neurobiologiae Experimentalis

• Publication date

  2008-09-30

• Fields of study

  Biology, Medicine

• Identifiers
  DOI 10.55782/ane-2008-1709PMID 18668166
• External record

  Open on Semantic Scholar

• Source metadata

  Semantic Scholar, PubMed

• No claims are published for this paper.

• No concepts are published for this paper.

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