Combination of stem cell and gene therapy ameliorates symptoms in Huntington's disease mice.

Huntington's disease (HD) is a dominantly inherited monogenetic disorder characterized by motor and cognitive dysfunction due to neurodegeneration. The disease is caused by the polyglutamine (polyQ) expansion at the 5' terminal of the exon 1 of the huntingtin () gene, , which results in the accumulation of mutant HTT (mHTT) aggregates in neurons and cell death. The monogenetic cause and the loss of specific neural cell population make HD a suitable candidate for stem cell and gene therapy.