Therapeutic potential of RNA interference for neurological disorders - RNA

Therapeutic potential of RNA interference for neurological disorders
Received 21 March 2006; accepted 2 June 2006. Available online 15 June 2006.
Dinah W.Y. Saha
Life Sciences
Volume 79, Issue 19 , 4 October 2006
ScienceDirect
Copyright ? 2006 Elsevier Inc. All rights reserved.
aAlnylam Pharmaceuticals, Inc., 300 Third Street, Cambridge, MA 02142, United States
Abstract
During the past decade, numerous molecular mediators of neurodegenerative diseases and neurological disorders have been identified and validated, yet few novel therapies have emerged and the unmet medical needs remain high. These molecular mediators belong to target classes such as ion channels, neurotransmitters and neurotransmitter receptors, cytokines, growth factors, enzymes and other proteins. In some cases, substantial pre-clinical validation exists, but the molecular target has not been readily druggable with small molecules, proteins or antibodies. RNA interference represents a therapeutic approach applicable to such non-druggable targets. Both non-viral and viral delivery strategies are being undertaken for in vivo silencing of molecular targets by RNA interference, which has resulted in robust efficacy in animal models of Alzheimer's disease, ALS, Huntington's disease, spinocerebellar ataxia, anxiety, depression, neuropathic pain, encephalitis and glioblastoma. These proof-of-concept data in animal models, together with the commencement of clinical trials using RNA interference for macular degeneration and respiratory syncytial virus infection, point to the potential of direct RNA interference for neurological disorders and neurodegenerative diseases.
Keywords: RNA interference; Nervous system; Drug development

Conflict of interest?employee of Alnylam Pharmaceuticals.
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