THERAPEUTIC STRATEGIES FOR HUNTINGTONS-DISEASE BASED ON A MOLECULAR UNDERSTANDING OF THE DISORDER

A mutation on chromosome 4p16.3 with an expanded polyglutamine tract h as been identified as the cause of Huntington's disease (HD). The neur oscience and clinical community now faces the formidable challenge of using this information to develop a treatment against this fatal and c urrently untreatable disease. This article reviews the recent literatu re pertaining to HD and presents an overview of possible intervention strategies against the neurodegenerative process of HD. Because little is known about the physiological function of the HD gene, there are f our biological levels at which therapies could be devised. Identificat ion and cloning of the gene might direct novel therapies for HD using the following strategies: interference (1) at the DNA or (2) at the RN A level; (3) blocking the deleterious effect of the protein; and (4) p hysiological intervention using pharmacological agents or neural cell transplants.