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Stem cells in human neurodegenerative disorders — time for clinical translation?
Olle Lindvall, Zaal Kokaia
Olle Lindvall, Zaal Kokaia
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Stem cells in human neurodegenerative disorders — time for clinical translation?

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Abstract

Stem cell–based approaches have received much hype as potential treatments for neurodegenerative disorders. Indeed, transplantation of stem cells or their derivatives in animal models of neurodegenerative diseases can improve function by replacing the lost neurons and glial cells and by mediating remyelination, trophic actions, and modulation of inflammation. Endogenous neural stem cells are also potential therapeutic targets because they produce neurons and glial cells in response to injury and could be affected by the degenerative process. As we discuss here, however, significant hurdles remain before these findings can be responsibly translated to novel therapies. In particular, we need to better understand the mechanisms of action of stem cells after transplantation and learn how to control stem cell proliferation, survival, migration, and differentiation in the pathological environment.

Authors

Olle Lindvall, Zaal Kokaia

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Figure 3

Stem cell–based therapies for AD.

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Stem cell–based therapies for AD.
AD leads to neuronal loss in the basal...
AD leads to neuronal loss in the basal forebrain cholinergic system, amygdala, hippocampus, and cortical areas of the brain; formation of neurofibrillary tangles; and β-amyloid protein accumulation in senile plaques. Stem cell–based therapy could be used to prevent progression of the disease by transplanting stem cells modified to release growth factors. Alternatively, compounds and/or antibodies could be infused to restore impaired hippocampal neurogenesis.

Copyright © 2026 American Society for Clinical Investigation
ISSN: 0021-9738 (print), 1558-8238 (online)

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