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Brain Nerve. 2007 Oct;59(10):1187-94.

[Gene therapy of ALS with short interfering RNA].

[Article in Japanese]

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Department of Neurology and Neurological Science, Tokyo Medical and Dental University, 1-5-45 Yushima, Bunkyo-ku, Tokyo 113-8519, Japan.


RNA 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 syste. 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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