Reference - Detail
| RRC ID | 41340 |
|---|---|
| Author | Tanaka K, Yamada T, Ohyagi Y, Asahara H, Horiuchi I, Kira J. |
| Title | Suppression of transthyretin expression by ribozymes: a possible therapy for familial amyloidotic polyneuropathy. |
| Journal | 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. |
| Volume | 183(1) |
| Pages | 79-84 |
| Published | 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 |
| Times Cited | 23 |
| WOS Category | CLINICAL NEUROLOGY NEUROSCIENCES |
| Altmetric score |
オルトメトリクス指標項目
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| The most frequently cited source | Patent(IFI CLAIMS) |
| Total number of mentions | 6 |
| Altmetric score changes over past 6months | 3.0 |
| Resource | |
| Human and Animal Cells | Hep G2(RCB0459) |