Ribonucleic acid interference for neurological disorders: candidate diseases, potential targets, and current approaches.
OBJECTIVE
Ribonucleic acid (RNA) interference (RNAi) is a conserved evolutionary defense mechanism that is gaining utility for therapeutic application by modulating gene expression or silencing disease-causing genes.


METHODS
This strategy has recently achieved success in mammalian cells via synthetic small interfering RNA or short hairpin RNA expressed in vectors for gene delivery. The vector-based RNAi strategy has particular potential because of the possibility of targeted gene delivery, long-term gene expression, and the potential means of penetrating the blood-brain barrier.


RESULTS
RNAi-based approaches have been proposed for a variety of neurological disorders, including dominant genetic diseases, neurodegenerative diseases, malignant brain tumors, pain, and viral-induced encephalopathies.


CONCLUSIONS
This review summarizes the current approaches of the RNAi strategy for neurological disorders, focusing on potential targets for therapeutic intervention.
Federici
Thais
T
Department of Neuroscience The Cleveland Clinic Foundation, Cleveland, Ohio 44195, USA.
Boulis
Nicholas M
NM
D018390:Gene Targeting / Q000379:methods* / Q000639:trends; D015316:Genetic Therapy / Q000379:methods / Q000639:trends; D006801:Humans; D009422:Nervous System Diseases / Q000235:genetics* / Q000628:therapy*; D034622:RNA Interference