RNAi Silencing in Mouse Models of Neurodegenerative Diseases

Title: RNAi Silencing in Mouse Models of Neurodegenerative Diseases

Volume: 4 Issue: 2

Author(s): Mohamed H. Farah

Affiliation:

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

Abstract: 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.

Cite this article as:

Farah H. Mohamed, RNAi Silencing in Mouse Models of Neurodegenerative Diseases, Current Drug Delivery 2007; 4 (2) . https://dx.doi.org/10.2174/156720107780362276