RRC ID 68702
著者 Hamasaki T, Suzuki H, Shirohzu H, Matsumoto T, D'Alessandro-Gabazza CN, Gil-Bernabe P, Boveda-Ruiz D, Naito M, Kobayashi T, Toda M, Mizutani T, Taguchi O, Morser J, Eguchi Y, Kuroda M, Ochiya T, Hayashi H, Gabazza EC, Ohgi T.
タイトル Efficacy of a novel class of RNA interference therapeutic agents.
ジャーナル PLoS One
Abstract RNA interference (RNAi) is being widely used in functional gene research and is an important tool for drug discovery. However, canonical double-stranded short interfering RNAs are unstable and induce undesirable adverse effects, and thus there is no currently RNAi-based therapy in the clinic. We have developed a novel class of RNAi agents, and evaluated their effectiveness in vitro and in mouse models of acute lung injury (ALI) and pulmonary fibrosis. The novel class of RNAi agents (nkRNA®, PnkRNA™) were synthesized on solid phase as single-stranded RNAs that, following synthesis, self-anneal into a unique helical structure containing a central stem and two loops. They are resistant to degradation and suppress their target genes. nkRNA and PnkRNA directed against TGF-β1mRNA ameliorate outcomes and induce no off-target effects in three animal models of lung disease. The results of this study support the pathological relevance of TGF-β1 in lung diseases, and suggest the potential usefulness of these novel RNAi agents for therapeutic application.
巻・号 7(8)
ページ e42655
公開日 2012-1-1
DOI 10.1371/journal.pone.0042655
PII PONE-D-12-09301
PMID 22916145
PMC PMC3419724
MeSH Acute Lung Injury / therapy* Animals Base Sequence Bronchoalveolar Lavage Fluid Disease Models, Animal Mice Pulmonary Fibrosis / therapy* RNA Interference* RNA, Messenger / genetics RNA, Small Interfering Transforming Growth Factor beta1 / genetics
IF 2.74
リソース情報
ヒト・動物細胞 Hepa 1-6(RCB1638)