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Therapeutic Potential of Small Interfering RNA for Central Nervous System Diseases
Amy E. Lovett-Racke, PhD;
Petra D. Cravens, PhD;
Anne R. Gocke, BS;
Michael K. Racke, MD;
Olaf Stüve, MD, PhD
Arch Neurol. 2005;62:1810-1813.
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INTRODUCTION
RNA interference (RNAi) is an evolutionarily conserved mechanism to degrade messenger RNA (mRNA) that has sequence homology to small double-stranded RNA (dsRNA). It is believed that the normal biological function of RNAi is to protect the host cells from RNA viruses and transposons, which can jeopardize the genome. Production of dsRNA in the host cell signals a series of events that ultimately results in the degradation of complementary mRNA. Recently, small interfering RNA (siRNA) has been used to study the function and significance of a vast number of genes in a variety of cell types. In the future, siRNA may have tremendous potential as gene-specific therapeutic agents for the treatment of many diseases. We discuss the potential role of siRNA as a novel therapeutic strategy for several central nervous system . . . [Full Text of this Article]
MECHANISMS OF RNAi
SPINOCEREBELLAR ATAXIA AND OTHER POLYGLUTAMINE-ASSOCIATED ATAXIAS
ALZHEIMER DISEASE
MULTIPLE SCLEROSIS
CNS TUMORS
CONCLUSIONS
AUTHOR INFORMATION
Author Affiliations: Department of Neurology, UT Southwestern Medical Center (Drs Lovett-Racke, Cravens, Racke, and Stüve and Ms Gocke), and Center for Immunology (Dr Racke), Dallas, Tex.
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