Perspectives

Nature Reviews Neuroscience 7, 75-84 (January 2006) | doi:10.1038/nrn1829

OpinionGene therapy: can neural stem cells deliver?

Franz-Josef Müller1,2, Evan Y. Snyder1 & Jeanne F. Loring1  About the authors

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Neural stem cells are a self-renewing population that generates the neurons and glia of the developing brain. They can be isolated, proliferated, genetically manipulated and differentiated in vitro and reintroduced into a developing, adult or pathologically altered CNS. Neural stem cells have been considered for use in cell replacement therapies in various neurodegenerative diseases, and an unexpected and potentially valuable characteristic of these cells has recently been revealed — they are highly migratory and seem to be attracted to areas of brain pathology such as ischaemic and neoplastic lesions. Here, we speculate on the ways in which neural stem cells might be exploited as delivery vehicles for gene therapy in the CNS.

Author affiliations

  1. Franz-Josef Müller, Evan Y. Snyder and Jeanne F. Loring are at the Burnham Institute for Medical Research, La Jolla, California, USA.
  2. Franz-Josef Müller is also at the Zentrum für Integrative Psychiatrie, Kiel, Germany.

Correspondence to: Franz-Josef Müller1,2 Email: fmueller@burnham.org

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