| 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
|
| Resource |
| Human and Animal Cells |
Hep G2(RCB0459) |
