RNAi Silencing in Mouse Models of Neurodegenerative Diseases

Author(s): Mohamed H. Farah

Journal Name: Current Drug Delivery

Volume 4 , Issue 2 , 2007

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RNA interference (RNAi) has emerged as a potential therapeutic approach for neurodegenerative diseases, particularly those associated with autosomal dominant patterns of inheritance. In proof of concept experiments, several groups have demonstrated efficacy of using viral vectors expressing short hairpin RNA (shRNA) directed against therapeutically relevant genes in mouse models of neurodegenerative diseases, including spinocerebellar ataxia, Amyotrophic Lateral Sclerosis, Huntington s Disease and amyloidosis (a pathological aspect of Alzheimers Disease). Although viralbased RNAi has limitations that most likely will preclude its usage in humans, a few recent developments underscore the potential of non-viral-based delivery of relevant RNAi as therapeutics for neurodegenerative diseases. Here, I will review the recent literature on effectiveness of RNAi as a therapeutic strategy in mouse models of neurodegenerative diseases.

Keywords: RNA-induced silencing complex (RISC), short hairpin RNA (shRNA), APP mouse model, secretases, Spinocerebellar ataxia type 1, Huntington Disease

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Article Details

Year: 2007
Page: [161 - 167]
Pages: 7
DOI: 10.2174/156720107780362276
Price: $65

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