Allele-specific RNAi selectively silences mutant SOD1 and achieves significant therapeutic benefit in vivo

Allele-specific RNAi selectively silences mutant SOD1 and achieves significant therapeutic benefit in vivo

RNA interference (RNAi) has the potential to treat diseases caused by dominant, gain-of-function type of gene mutations. In these diseases, one allele is mutated and produces a toxic protein, whereas the other allele is normal and performs vital functions. One challenge in the treatment is to specif...

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Bibliographic Details
Main Authors: Xugang Xia, Hongxia Zhou, Yong Huang, Zuoshang Xu
Format: Article
Language:English
Published: Elsevier 2006-09-01
Series:Neurobiology of Disease
Subjects:
Neurodegenerative disease
Neurodegeneration
Dominant
Gain-of-function
RNAi therapy
Transgenic mice
Online Access:http://www.sciencedirect.com/science/article/pii/S0969996106001021

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http://www.sciencedirect.com/science/article/pii/S0969996106001021

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