[HTML][HTML] Cellular therapy and induced neuronal replacement for Huntington's disease

A Benraiss, SA Goldman - Neurotherapeutics, 2011 - Elsevier
Huntington's disease (HD) is an inherited, relentlessly progressive neurodegenerative
disease with an invariably fatal outcome. HD is inherited in an autosomal dominant fashion,
and is characterized pathologically by the loss of cortical and striatal neurons, and clinically
by involuntary choreiform movements accompanied by progressive cognitive impairment
and emotional lability. The disorder is caused by an expanded cystosine adenine guanine
(CAG) tri-nucleotide repeat encoding polyglutamine (polyQ) in the first exon of the Huntingtin …