Progress and challenges in RNA interference therapy for Huntington disease.
Huntington disease is an incurable, dominant neurodegenerative disorder caused by polyglutamine repeat expansion in the huntingtin protein. Reducing mutant huntingtin expression may offer a treatment for Huntington disease. RNA interference has emerged as a powerful method to silence dominant disease genes. As such, it is being developed as a prospective Huntington disease therapy. Here I discuss the current progress and important remaining challenges of RNA interference therapy for Huntington disease.
Harper
Scott Q
SQ
Department of Pediatrics, The Ohio State University Medical Center, and Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, 700 Children's Dr, Room WA2015, Columbus, OH 43205, USA. harper.104@osu.edu
D000818:Animals; D020029:Base Pairing / Q000235:genetics; D004195:Disease Models, Animal; D020868:Gene Silencing; D005799:Genes, Dominant / Q000235:genetics; D015316:Genetic Therapy; D005822:Genetic Vectors / Q000235:genetics / Q000627:therapeutic use; D006801:Humans; D000071058:Huntingtin Protein; D006816:Huntington Disease / Q000235:genetics* / Q000628:therapy*; D051379:Mice; D035683:MicroRNAs / Q000235:genetics; D009419:Nerve Tissue Proteins / Q000235:genetics*; D009687:Nuclear Proteins / Q000235:genetics*; D011446:Prospective Studies; D034622:RNA Interference / Q000502:physiology*; D012333:RNA, Messenger / Q000235:genetics; D034743:RNA-Induced Silencing Complex / Q000235:genetics; D014162:Transfection / Q000379:methods
C086055:HTT protein, human; D000071058:Huntingtin Protein; D035683:MicroRNAs; D009419:Nerve Tissue Proteins; D009687:Nuclear Proteins; D012333:RNA, Messenger; D034743:RNA-Induced Silencing Complex