[Gene therapy of ALS with short interfering RNA].
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.
Yokota
Takanori
T
Department of Neurology and Neurological Science, Tokyo Medical and Dental University, 1-5-45 Yushima, Bunkyo-ku, Tokyo 113-8519, Japan.
D000690:Amyotrophic Lateral Sclerosis / Q000235:genetics / Q000628:therapy*; D000818:Animals; D002490:Central Nervous System; D004195:Disease Models, Animal; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors / Q000235:genetics; D006801:Humans; D034622:RNA Interference; D012330:RNA, Double-Stranded; D034741:RNA, Small Interfering / Q000008:administration & dosage*; D013482:Superoxide Dismutase / Q000235:genetics; D000072105:Superoxide Dismutase-1; D014780:Viruses / Q000235:genetics
D012330:RNA, Double-Stranded; D034741:RNA, Small Interfering; C000606290:SOD1 protein, human; C000606291:Sod1 protein, mouse; D013482:Superoxide Dismutase; D000072105:Superoxide Dismutase-1