Lysosomal Proteins as a Therapeutic Target in Neurodegeneration

Jessica M. Mc Donald, Dimitri Krainc

Research output: Contribution to journalReview articlepeer-review

13 Scopus citations


Several proteins that are mutated in lysosomal storage diseases are linked to neurodegenerative disease. This review focuses on some of these lysosomal enzymes and transporters, as well as current therapies that have emerged from the lysosomal storage disease field. Given the deeper genetic understanding of lysosomal defects in neurodegeneration, we explore why some of these orphan disease drug candidates are also attractive targets in subpopulations of individuals with neurodegenerative disease.

Original languageEnglish (US)
Pages (from-to)445-458
Number of pages14
JournalAnnual review of medicine
StatePublished - Jan 14 2017


  • Lysosomal storage disease
  • Neurodegenerative disease
  • Neurodegenerative drug candidates
  • Parkinson's disease

ASJC Scopus subject areas

  • Biochemistry, Genetics and Molecular Biology(all)

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