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

• Authors: C. Pierret , J.A. Morrison , M.D. Kirk
• Languages of publication: EN

Abstracts

EN

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.

Keywords

EN

GENE THERAPY; LYSOSOMAL STORAGE; STEM CELL

Discipline

• EXPERIMENTAL_BIOLOGY_&_MEDICINE: EXPERIMENTAL BIOLOGY & MEDICINE

• Publisher: Marceli Nencki Institute of Experimental Biology, Polish Academy of Sciences
• Journal: Acta Neurobiologiae Experimentalis
• Year: 2008
• Volume: 68
• Issue: 3
• Pages: 429-442

Contributors

author

C. Pierret

author

J.A. Morrison

author

M.D. Kirk

• Document Type: REVIEW
• Publication order reference: Mark D. Kirk, Division of Biological Sciences, University of Missouri, Columbia, MO 65211, USA