Gene therapy for Huntington's disease

• Main Authors: Shilpa Ramaswamy, Jeffrey H. Kordower
• Format: Article
• Language: English
• Published: Elsevier 2012-11-01
• Series: Neurobiology of Disease
• Subjects: Gene therapy; Adeno-associated viral (AAV) vectors; Serotype; Pseudotypes; Brain-derived neurotrophic factor (BDNF); Ciliary neurotrophic factor (CNTF)
• Online Access: http://www.sciencedirect.com/science/article/pii/S0969996111004050

Huntington's disease (HD) is a neurodegenerative disease for which there is no cure. Therapies that are efficacious in animal models have to date shown benefit for humans. One potential powerful approach is gene therapy. The ideal method of administration of gene therapy has been hotly debated and viral vectors have provided one method of long-term and wide-spread delivery to the brain. Trophic factors to protect cells from degeneration and RNAi to reduce mutant huntingtin (mHtt) protein expression are 2 main classes of compounds that demonstrate benefit in animal models. This review will examine some commonly used adeno-associated viral (AAV) vectors and discuss some therapies that hold promise for HD.