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 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 language | English (US) |
|---|---|
| Pages (from-to) | 429-442 |
| Number of pages | 14 |
| Journal | Acta Neurobiologiae Experimentalis |
| Volume | 68 |
| Issue number | 3 |
| State | Published - 2008 |
Keywords
- Enzyme replacement
- Gene therapy
- Lysosomal storage
- Neuronal ceroid-lipofuscinoses
- Stem cell
- Substrate reduction
ASJC Scopus subject areas
- General Neuroscience