Established and emerging therapies for Huntington's disease.
Huntington's disease is a genetic, neurodegenerative disorder causing cell dysfunction prior to cell death. Mechanisms that underlie the pathological changes continue to be elucidated, which in turn increases the number of potential therapeutic targets which have the ability to reverse or prevent further cell damage. As well as cell protection strategies, cell replacement techniques have been developed with the aim of replacing dead cells and restoring functional circuits. This review describes therapies used in clinical practice, therapies that have shown promise in experimental models either at the genetic or molecular level, and therapies that are subject to human clinical trials. It is likely that any successful therapy in clinical practice will involve a number of different approaches aimed at different targets in order to achieve both cell protection and cell replacement.
Wright
Ben L C
BL
Cambridge Centre for Brain Repair, E.D. Adrian Building, Forvie Site, Cambridge, UK. blcw2@cam.ac.uk
Barker
Roger A
RA
D000818:Animals; D017690:Cell Transplantation / Q000639:trends; D019610:Cytoprotection / Q000187:drug effects; D006801:Humans; D006816:Huntington Disease / Q000188:drug therapy / Q000517:prevention & control / Q000628:therapy*