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Cell Stem Cell. 2012 Apr 6;10(4):455-64. doi: 10.1016/j.stem.2012.01.021. Epub 2012 Mar 15.

Human embryonic stem cell-derived GABA neurons correct locomotion deficits in quinolinic acid-lesioned mice.

Author information

1
Department of Anatomy, Histology & Embryology, Shanghai Medical College, Fudan University, China.

Abstract

Degeneration of medium spiny GABA neurons in the basal ganglia underlies motor dysfunction in Huntington's disease (HD), which presently lacks effective therapy. In this study, we have successfully directed human embryonic stem cells (hESCs) to enriched populations of DARPP32-expressing forebrain GABA neurons. Transplantation of these human forebrain GABA neurons and their progenitors, but not spinal GABA cells, into the striatum of quinolinic acid-lesioned mice results in generation of large populations of DARPP32(+) GABA neurons, which project to the substantia nigra as well as receiving glutamatergic and dopaminergic inputs, corresponding to correction of motor deficits. This finding raises hopes for cell therapy for HD.

PMID:
22424902
PMCID:
PMC3322292
DOI:
10.1016/j.stem.2012.01.021
[Indexed for MEDLINE]
Free PMC Article

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