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

Journal of the Neurological Sciences

Volumn 183, Issue 1, 2001, Pages 79-84

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

^a KYUSHU UNIVERSITY   (Japan)

Author keywords

Familial amyloidotic polyneuropathy; Gene therapy; Ribozymes; Transthyretin

Indexed keywords

PREALBUMIN; RIBOZYME;

AMYLOID NEUROPATHY; ARTICLE; AUTOSOMAL DOMINANT INHERITANCE; CELL STRAIN COS1; GENE MUTATION; GENE THERAPY; GENETIC TRANSCRIPTION; GENETIC TRANSFECTION; HUMAN; HUMAN CELL; IMMUNOBLOTTING; NUCLEOTIDE SEQUENCE; PRIORITY JOURNAL; PROTEIN DEGRADATION; PROTEIN EXPRESSION;

AMYLOID NEUROPATHIES; DOWN-REGULATION; GENE THERAPY; HUMANS; PREALBUMIN; RNA, CATALYTIC; RNA, MESSENGER;

EID: 0035862631     PISSN: 0022510X     EISSN: None     Source Type: Journal    
DOI: 10.1016/S0022-510X(00)00481-0     Document Type: Article

References (14)

1. Amyloidosis and neuropathy
2. Outcome of liver transplantation for transthyretin amyloidosis: follow-up of Japanese familial amyloidotic polyneuropathy patients
3. Sequence requirements of the hammerhead RNA self-cleavage reaction
4. Specificity of the hairpin ribozyme
5. A basic transthyretin variant (Glu61>Lys) causes familial amyloidotic polyneuropathy: protein and DNA sequencing and PCR-induced mutation restriction analysis
6. Retinol binding protein and transthyretin are secreted as a complex formed in the endoplasmic reticulum in HepG2 human hepatocarcinoma cells
7. Delivery of a hammerhead ribozyme specifically down-regulates the production of fibrillin-1 by cultured dermal fibroblasts
8. Diagnosis of familial amyloidotic polyneuropathy by recombinant DNA techniques
9. 15.1 hammerhead ribozymes for targeting the transthyretin-30 mutation
10. A general purpose RNA-cleaving DNA enzyme
11. Receptor-mediated gene delivery in vivo
12. Receptor-mediated transfer of pSV2CAT DNA to mouse liver cells using asialofetuin-labeled liposome
13. Progressive cardiac amyloidosis following liver transplantation for familial amyloid polyneuropathy
14. Disruption of the transthyretin gene results in mice with depressed levels of plasma retinol and thyroid hormone