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

Lixiang Ma, Baoyang Hu, Yan Liu, Scott Christopher Vermilyea, Huisheng Liu, Lu Gao, Yan Sun, Xiaoqing Zhang, Su Chun Zhang

Research output: Contribution to journalArticlepeer-review

252 Scopus citations

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.

Original languageEnglish (US)
Pages (from-to)455-464
Number of pages10
JournalCell Stem Cell
Volume10
Issue number4
DOIs
StatePublished - Apr 6 2012
Externally publishedYes

Fingerprint

Dive into the research topics of 'Human embryonic stem cell-derived GABA neurons correct locomotion deficits in quinolinic acid-lesioned mice'. Together they form a unique fingerprint.

Cite this