Biochemical and biophysical research communications
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Biochem. Biophys. Res. Commun. · Nov 2005
Selective silencing of a mutant transthyretin allele by small interfering RNAs.
Familial amyloidotic polyneuropathy (FAP) is a hereditary systemic amyloidosis caused by dominantly acting missense mutations in the gene encoding transthyretin (TTR). The most common mutant TTR is of the Val30Met type, which results from a point mutation. ⋯ We have identified an siRNA that specifically inhibits mutant, but not wild-type, TTR expression even in cells expressing both alleles. Thus, this siRNA-based approach may have potential for the gene therapy of FAP.