TY - JOUR
T1 - RNAi silencing in mouse models of neurodegenerative diseases
AU - Farah, Mohamed H.
PY - 2007/4
Y1 - 2007/4
N2 - 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 Alzheimer's Disease). Although viral-based 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.
AB - 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 Alzheimer's Disease). Although viral-based 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.
UR - http://www.scopus.com/inward/record.url?scp=34147183745&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=34147183745&partnerID=8YFLogxK
U2 - 10.2174/156720107780362276
DO - 10.2174/156720107780362276
M3 - Review article
C2 - 17456035
AN - SCOPUS:34147183745
SN - 1567-2018
VL - 4
SP - 161
EP - 167
JO - Current Drug Delivery
JF - Current Drug Delivery
IS - 2
ER -