Rinsho Shinkeigaku (Clinical Neurology)

The 50th Annual Meeting of the Japanese Society of Neurology

Gene therapy of ALS with RNA interference

Takanori Yokota, M.D.

Department of Neurology and Neurological Science, Tokyo Medical and Dental University

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 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.
Full Text of this Article in Japanese PDF (201K)

(CLINICA NEUROL, 49: 821|823, 2009)
key words: small-interfering RNA, short-hairpin RNA, AAV, gene therapy, RNA interference

(Received: 21-May-09)