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RNAi silencing in mouse models of neurodegenerative diseases.
Curr Drug Deliv. 2007 Apr; 4(2):161-7.CD

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 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.

Authors+Show Affiliations

Division of Neuropathology, Department of Pathology, Johns Hopkins University School of Medicine, Baltimore, MD 21205-2196, USA. mfarah2@jhem.jhmi.edu

Pub Type(s)

Journal Article
Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't
Review

Language

eng

PubMed ID

17456035

Citation

Farah, Mohamed H.. "RNAi Silencing in Mouse Models of Neurodegenerative Diseases." Current Drug Delivery, vol. 4, no. 2, 2007, pp. 161-7.
Farah MH. RNAi silencing in mouse models of neurodegenerative diseases. Curr Drug Deliv. 2007;4(2):161-7.
Farah, M. H. (2007). RNAi silencing in mouse models of neurodegenerative diseases. Current Drug Delivery, 4(2), 161-7.
Farah MH. RNAi Silencing in Mouse Models of Neurodegenerative Diseases. Curr Drug Deliv. 2007;4(2):161-7. PubMed PMID: 17456035.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - RNAi silencing in mouse models of neurodegenerative diseases. A1 - Farah,Mohamed H, PY - 2007/4/26/pubmed PY - 2007/5/18/medline PY - 2007/4/26/entrez SP - 161 EP - 7 JF - Current drug delivery JO - Curr Drug Deliv VL - 4 IS - 2 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. SN - 1567-2018 UR - https://www.unboundmedicine.com/medline/citation/17456035/RNAi_silencing_in_mouse_models_of_neurodegenerative_diseases_ L2 - https://www.ingentaconnect.com/openurl?genre=article&issn=1567-2018&volume=4&issue=2&spage=161&aulast=Farah DB - PRIME DP - Unbound Medicine ER -