Cell-based therapies for Huntington's disease.
Atlanta, United States. In Drug Discov Today, Jul 2014
Current research focuses on genetic suppression of the mutant huntingtin (mHTT) gene and cell replacement therapy of the lost cells in HD.
Milano, Italy. In Handb Exp Pharmacol, Dec 2013
However, only in Huntington disease (HD) gain- and loss-of-function experiments have mechanistically linked these abnormalities with the genetic defect.In this chapter we will describe how huntingtin protein, whose mutation causes HD, is involved in the physiological control of BDNF synthesis and transport in neurons and how both processes are simultaneously disrupted in HD.