Nerve Injection of Viral Vectors Efficiently Transfers Transgenes into Motor Neurons and Delivers RNAi Therapy Against ALS

RNA interference (RNAi) mediates sequence-specific gene silencing, which can be harnessed to silencing disease-causing genes for therapy. Particularly suitable diseases are those caused by dominant, gain-of-function type of gene mutations. In these diseases, the mutant gene generates a mutant protei...

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Main Authors: Wu, Rui, Wang, Hongyan, Xia, Xugang, Zhou, Hongxia, Liu, Chunyan, Castro, Maria, Xu, Zuoshang
Format: Artigo
Jezik:Inglês
Izdano: Mary Ann Liebert, Inc. 2009
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Forum Original Research Communications
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC2842583/
https://ncbi.nlm.nih.gov/pubmed/19344276
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/ars.2009.2618
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