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The Emerging Therapeutic Role of RNA Interference in Disorders of the Central Nervous System
Author(s) -
Dessy A,
Gorman JM
Publication year - 2011
Publication title -
clinical pharmacology and therapeutics
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.941
H-Index - 188
eISSN - 1532-6535
pISSN - 0009-9236
DOI - 10.1038/clpt.2010.312
Subject(s) - central nervous system , clinical pharmacology , neuroscience , rna interference , medicine , rna , biology , pharmacology , genetics , gene
Several challenges have been encountered in the attempt to apply RNA interference (RNAi) to the treatment of disease, including lack of effective delivery, off‐target responses, and instability of reagents. The treatment of neuropsychiatric disorders poses additional obstacles, given that drugs have only limited access to the central nervous system (CNS) and that the neurons are in a postmitotic state. Despite these inherent limitations, much progress has been made toward attaining a better understanding of diseases of the CNS, and the development of effective treatments appears to be a distinct possibility in the near future. Clinical Pharmacology & Therapeutics (2011) 89 3, 450–454. doi: 10.1038/clpt.2010.312