Neural Stem Cell Therapy in Lysosomal Storage Disorders

  • Jean-Pyo Lee
  • Dan Clark
  • Mylvaganam Jeyakumar
  • Rodolfo Gonzalez
  • Scott Mckercher
  • Franz-Josef Muller
  • Rahul Jandial
  • Rosanne M. Taylor
  • Kook In Park
  • Thomas N. Seyfried
  • Frances M. Platt
  • Evan Y. Snyder

Many lysosomal storage disorders (LSDs) produce neurodegeneration as a prominent feature (Neufeld, 1991). LSDs are autosomal recessive metabolic diseases caused by deficiencies of specific acid hydrolases resulting in accumulation of unmetabolized substrates and macromolecules in lysosomes. There are ~50 diseases that can be classified as LSDs. The precise mechanisms underlying the actual neurodegenerative process remain to be determined, however, it is known that replacement of the absent gene product typically restores normal metabolism to a cell including forestalling neural cell dysfunction, at least in vitro. Nevertheless, there are currently no effective treatments for the neurological manifestations of the infantile-onset forms of the LSDs. The neuropathology of LSDs is characterized not by discrete focal neuropathology, as in Parkinson’s disease, but rather by extensive, multifocal, or even “global” neural degeneration or dysfunction. Therapy may require not only therapeutic molecules, such as enzymes, but also widespread neural cell replacement.

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Copyright information

© Springer Science+Business Media, LLC 2007

Authors and Affiliations

  • Jean-Pyo Lee
    • 1
  • Dan Clark
    • 2
  • Mylvaganam Jeyakumar
    • 3
  • Rodolfo Gonzalez
    • 4
  • Scott Mckercher
    • 5
  • Franz-Josef Muller
    • 5
  • Rahul Jandial
    • 6
  • Rosanne M. Taylor
    • 7
  • Kook In Park
    • 8
  • Thomas N. Seyfried
    • 9
  • Frances M. Platt
    • 3
  • Evan Y. Snyder
    • 1
  1. 1.The Burnham Institute for Medical Research, and Department of PediatricsUniversity of California San DiegoLa JollaUSA
  2. 2.Department of Medical GeneticsUniversity of British Columbia, Child and Family Research InstituteVancouverCanada
  3. 3.Department of Biochemistry, Glycobiology InstituteUniversity of OxfordUK
  4. 4.Center for Neuroscience and Aging ResearchBurnham Institute for Medical ResearchLa JollaUSA
  5. 5.Burnham Institute for Medical ResearchLa JollaUSA
  6. 6.Department of NeurosurgeryUCSD School of MedicineLa JollaUSA
  7. 7.Department of Animal Science, Faculty of Veterinary ScienceUniversity of SydneyAustralia
  8. 8.Department of PediatricsYonsei University College of MedicineKorea
  9. 9.Department of BiologyBoston CollegeChestnut HillUSA

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