Journal
TRENDS IN MOLECULAR MEDICINE
Volume 18, Issue 11, Pages 667-678Publisher
ELSEVIER SCI LTD
DOI: 10.1016/j.molmed.2012.09.004
Keywords
aminoglycosides; hereditary cancer syndromes; nonsense-suppression; PTC124; readthrough; suppressor-tRNA; nonsense-mediated decay (NMD); premature termination codon (PTC)
Funding
- Calouste Gulbenkian Foundation
- Portuguese Foundation for Science and Technology (FCT) [SFRH/BD/4646212008-RBC]
- European Social Fund and Portuguese Ministry of Science and Technology (MCTES) [POPH - QREN/Type 4.2]
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Several hereditary cancer syndromes are associated with nonsense mutations that create premature termination codons (PTC). Therapeutic strategies involving readthrough induction partially restore expression of proteins with normal function from nonsense-mutated genes, and small molecules such as aminoglycosides and PTC124 have exhibited promising results for treating patients with cystic fibrosis and Duchenne muscular dystrophy. Transgenic expression of suppressor-tRNAs and depleting translation termination factors are, among others, potential strategies for treating PTC-associated diseases. In this review, the potential of using readthrough strategies as a therapy for cancer syndromes is discussed, and we consider the effect of nonsense-mediated decay and other factors on readthrough efficiency.
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