Embryonic stem cell-derived GABA progenitor cells for treatment of Huntington's disease

Transplantation of ESC-derived human forebrain GABA neurons and their progenitor 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 and receive glutamatergic and dopaminergic inputs, corresponding to correction of motor deficits. These findings raise hopes for cell therapy for Huntington disease.