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

19Citations
Citations of this article
26Readers
Mendeley users who have this article in their library.

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. © 2008 by Polish Neuroscience Society - PTBUN, Nencki Institute of Experimental Biology.

References Powered by Scopus

The biogenesis of lysosomes

1439Citations
N/AReaders
Get full text

Convection-enhanced delivery of macromolecules in the brain

1260Citations
N/AReaders
Get full text

Enzyme replacement therapy in fabry disease a randomized controlled trial

1204Citations
N/AReaders
Get full text

Cited by Powered by Scopus

Clinical trials in rare disease: Challenges and opportunities

181Citations
N/AReaders
Get full text

Ceria nanoparticles stabilized by organic surface coatings activate the lysosome-autophagy system and enhance autophagic clearance

109Citations
N/AReaders
Get full text

2-Hydroxypropyl-β-cyclodextrin promotes transcription factor EB-mediated activation of autophagy: Implications for therapy

89Citations
N/AReaders
Get full text

Register to see more suggestions

Mendeley helps you to discover research relevant for your work.

Already have an account?

Cite

CITATION STYLE

APA

Pierret, C., Morrison, J. A., & Kirk, M. D. (2008). Treatment of lysosomal storage disorders: Focus on the neuronal ceroid-lipofuscinoses. Acta Neurobiologiae Experimentalis. https://doi.org/10.55782/ane-2008-1709

Readers' Seniority

Tooltip

PhD / Post grad / Masters / Doc 12

60%

Researcher 7

35%

Professor / Associate Prof. 1

5%

Readers' Discipline

Tooltip

Medicine and Dentistry 9

43%

Agricultural and Biological Sciences 6

29%

Biochemistry, Genetics and Molecular Bi... 4

19%

Engineering 2

10%

Article Metrics

Tooltip
Social Media
Shares, Likes & Comments: 1

Save time finding and organizing research with Mendeley

Sign up for free