title: RNAi-based therapies for Huntington's disease: delivery challenges and opportunities.
abstract: Huntington's disease (HD) is a polyglutamine neurodegenerative disease caused by a mutation in the HTT gene coding for the Huntingtin protein (HTT). Unfortunately, there is no cure for HD and there is also no known way to modify the disease progression. RNAi approaches offer the promise of a certain degree of control over the disease. However, there are several challenges in potential use of RNAi in the treatment of HD. This article will discuss the details of RNAi technology as applied to the treatment of HD, and novel approaches to overcome the drug delivery challenges.
authors.affiliation: Department of Pharmaceutica Sciences, College of Pharmacy & Health Sciences, Butler University, Indianapolis, IN 46208, USA.
authors.forename: Neelima
authors.initials: N
authors.lastname: Mantha
authors.forename: Sudip K
authors.initials: SK
authors.lastname: Das
authors.forename: Nandita G
authors.initials: NG
authors.lastname: Das
mesh_terms: D017209:Apoptosis; D005786:Gene Expression Regulation; D018014:Gene Transfer Techniques; D006801:Humans; D000071058:Huntingtin Protein; D006816:Huntington Disease / Q000473:pathology / Q000628:therapy*; D009419:Nerve Tissue Proteins / Q000502:physiology; D034622:RNA Interference; D034741:RNA, Small Interfering / Q000008:administration & dosage / Q000235:genetics
chemical_list: C086055:HTT protein, human; D000071058:Huntingtin Protein; D009419:Nerve Tissue Proteins; D034741:RNA, Small Interfering