RRC ID 41340
著者 Tanaka K, Yamada T, Ohyagi Y, Asahara H, Horiuchi I, Kira J.
タイトル Suppression of transthyretin expression by ribozymes: a possible therapy for familial amyloidotic polyneuropathy.
ジャーナル J Neurol Sci
Abstract Familial amyloidotic polyneuropathy type 1 (FAP) is an autosomal-dominantly inherited disorder with systemic deposition of a variant transthyretin (TTR). We attempted to suppress TTR production by ribozyme degradation of TTR mRNA. Hammerhead and hairpin ribozymes cleaved TTR mRNA at specific individual sites in vitro. A ribozyme targeting a variant TTR (E61K) degraded the variant mRNA, but not a wild-type mRNA. These ribozymes also reduced the amounts of TTR mRNA and protein in HepG2 cells and COS-1 cells transfected with TTR-E61K cDNA. Ribozymes might be studied further as a potential treatment for FAP.
巻・号 183(1)
ページ 79-84
公開日 2001-1-15
DOI 10.1016/s0022-510x(00)00481-0
PII S0022510X00004810
PMID 11166799
MeSH Amyloid Neuropathies / drug therapy Amyloid Neuropathies / metabolism* Down-Regulation / drug effects Down-Regulation / physiology Genetic Therapy / methods* Humans Prealbumin / drug effects* Prealbumin / metabolism RNA, Catalytic / pharmacology* RNA, Catalytic / therapeutic use RNA, Messenger / drug effects* RNA, Messenger / metabolism
IF 3.115
引用数 23
WOS 分野 CLINICAL NEUROLOGY NEUROSCIENCES
リソース情報
ヒト・動物細胞 Hep G2(RCB0459)