Gene therapy of Als with Rna interference

Yokota, T.

Rinsho Shinkeigaku 49(11): 821-823


ISSN/ISBN: 0009-918X
DOI: 10.5692/clinicalneurol.49.821
Accession: 068510684

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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.