Producing striatal phenotypes for transplantation in Huntington's disease.

Neural transplantation as a therapeutic strategy in neurodegenerative disorders offers to replace cells lost during the disease process, with the potential to reconstruct dysfunctional circuitry, thus alleviating associated disease symptoms. The focal loss of striatal cells, specifically medium-sized spiny neurons (MSN) in Huntington's disease (HD), makes transplantation a therapeutic option. Here, we review the progress made in generating striatal MSN phenotypes for transplantation in HD. We discuss the use of primary fetal tissue as a donor source in both preclinical and clinical studies and assess the options for renewable cell sources. We evaluate progress in directing the differentiation of renewable cells towards a striatal MSN phenotype for HD.