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.
RNAi silencing in mouse models of neurodegenerative diseases. Publishing Authors By Initials
RNAi silencing in mouse models of neurodegenerative diseases. Journal Published:
PUBLICATION TYPE: Review
Journal: Current drug delivery
VOLUME: 4
Page Numbers: 161-7
Journal Abbreviation:
ISSN: 1567-2018
DAY: 3
MONTH: Apr
YEAR: 2007
RNAi silencing in mouse models of neurodegenerative diseases. Information
Number of References:
LANGUAGE: eng
NlmUniqueID: 101208455
RNAi silencing in mouse models of neurodegenerative diseases. Keywords Mesh Terms:
KEYWORDS: Superoxide Dismutase
MESH TERMS: metabolism
Chemical & Substance for Abstract: RNAi silencing in mouse models of neurodegenerative diseases. Information
Substance Name: Bace1 protein, mouse
Registry Number: EC 3.4.23.46
Grant and Affiliation Information for RNAi silencing in mouse models of neurodegenerative diseases.
AFFILIATION: Division of Neuropathology, Department of Pathology, Johns Hopkins University School of Medicine, Baltimore, MD 21205-2196, USA. mfarah2@jhem.jhmi.edu
Country: United Arab Emirates
AGENCY: United States NIA
GRANT: AG025658
ACRONYM: AG
MEDLINETA: Curr Drug Deliv
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