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- Takanori Yokota.
- Department of Neurology and Neurological Science, Tokyo Medical and Dental University.
- Rinsho Shinkeigaku. 2009 Nov 1; 49 (11): 821-3.
AbstractRNA interference (RNAi) is the process of sequence-specific, post-tanscriptional gene silencing, initiated by double-stranded RNA (dsRNA). The gene therapy for familial ALS with siRNA had been started and showed promising results in the model mouse. There is a recent progress in the delivery of siRNA to the central nervous system. There are still important problems for application of gene therapy including off-target effect and gene delivery of siRNA, but a rapid progress can be expected because of the extremely high efficiency of siRNA.
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