Silencing neurodegenerative disease: bringing RNA interference to the clinic.
RNA interference (RNAi) is a recently described conserved biological pathway where non-coding RNAs suppress the expression of specific genes. Research efforts in the RNAi field aim to gain a better understanding of how its underlying machinery is orchestrated, to define the biological role of this conserved pathway, determine how to effectively manipulate RNAi in the laboratory and to integrate all this knowledge to develop novel therapies for human disease. This review summarizes the advances in the design of therapeutic RNAi for neurodegenerative diseases and discusses some of the experimental steps required to bring this therapy to human clinical trials.
Rodriguez-Lebron
Edgardo
E
Department of Neurology, Carver College of Medicine, University of Iowa, Iowa City, IA 52242-1101, USA. edgardo-rodriguez@mail.medicine.uiowa.edu
Gonzalez-Alegre
Pedro
P
D000818:Animals; D000067696:Ataxin-1; D000067528:Ataxins; D016503:Drug Delivery Systems; D005104:Expert Testimony; D006801:Humans; D009419:Nerve Tissue Proteins / Q000737:chemistry / Q000235:genetics / Q000627:therapeutic use; D019636:Neurodegenerative Diseases / Q000628:therapy*; D009687:Nuclear Proteins / Q000737:chemistry / Q000235:genetics / Q000627:therapeutic use; D034622:RNA Interference / Q000502:physiology*; D034741:RNA, Small Interfering / Q000008:administration & dosage* / Q000737:chemistry
C000593749:ATXN1 protein, human; D000067696:Ataxin-1; D000067528:Ataxins; D009419:Nerve Tissue Proteins; D009687:Nuclear Proteins; D034741:RNA, Small Interfering