Suppression of transthyretin expression by ribozymes: A possible therapy for familial amyloidotic polyneuropathy

Kimihiro Tanaka, Takeshi Yamada, Yasumasa Ohyagi, Hideaki Asahara, Izumi Horiuchi, Jun ichi Kira

Research output: Contribution to journalArticle

30 Citations (Scopus)

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.

Original languageEnglish
Pages (from-to)79-84
Number of pages6
JournalJournal of the Neurological Sciences
Volume183
Issue number1
DOIs
Publication statusPublished - Jan 15 2001

All Science Journal Classification (ASJC) codes

  • Neurology
  • Clinical Neurology

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