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Proc Natl Acad Sci U S A 1995 Aug 29;9218:8298-302.
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Toward the therapeutic editing of mutated RNA sequences.

Woolf TM , Chase JM , Stinchcomb DT .

If RNA editing could be rationally directed to mutated RNA sequences, genetic diseases caused by certain base substitutions could be treated. Here we use a synthetic complementary RNA oligonucleotide to direct the correction of a premature stop codon mutation in dystrophin RNA. The complementary RNA oligonucleotide was hybridized to a premature stop codon and the hybrid was treated with nuclear extracts containing the cellular enzyme double-stranded RNA adenosine deaminase. When the treated RNAs were translated in vitro, a dramatic increase in expression of a downstream luciferase coding region was observed. The cDNA sequence data are consistent with deamination of the adenosine in the UAG stop codon to inosine by double-stranded RNA adenosine deaminase. Injection of oligonucleotide-mRNA hybrids into Xenopus embryos also resulted in an increase in luciferase expression. These experiments demonstrate the principle of therapeutic RNA editing.

PubMed ID: 7545300
PMC ID: PMC41144
Article link: Proc Natl Acad Sci U S A

Species referenced: Xenopus
Genes referenced: ada ada.2 dmd dmd.2

References [+] :
Backus, Only cytidines 5' of the apolipoprotein B mRNA mooring sequence are edited. 1994, Pubmed