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

26 Mar 2019

Abstract excerpt

Abstract 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 (HTT) gene, IT15, which results in the accumulation of mutant HTT (mHTT) aggregates in neurons and cell death. The monogenetic cause and the loss...

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PMID: 30937182
DOI: 10.1038/s41536-019-0066-7

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