PMID: 7545300Aug 29, 1995Paper

Toward the therapeutic editing of mutated RNA sequences

Proceedings of the National Academy of Sciences of the United States of America
T M WoolfD T Stinchcomb

Abstract

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.

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Citations

Jun 7, 1991·Biochimica Et Biophysica Acta·D Richardson, E Baker
Nov 10, 2000·Advanced Drug Delivery Reviews·T Watanabe, B A Sullenger
Apr 29, 1998·Nature Biotechnology·T M Woolf
Apr 15, 1996·Nucleic Acids Research·S GryaznovB Calabretta
Oct 11, 2013·Proceedings of the National Academy of Sciences of the United States of America·Maria Fernanda Montiel-GonzalezJoshua J C Rosenthal
May 28, 2009·IUBMB Life·Yongfeng JinQi Li
Feb 11, 2015·Bioscience Reports·HuiQi HongLeilei Chen
Dec 20, 2011·Seminars in Cell & Developmental Biology·Leila E Rieder, Robert A Reenan
May 10, 2017·Bioscience Trends·Luyen Thi Vu, Toshifumi Tsukahara
Oct 3, 2003·Chembiochem : a European Journal of Chemical Biology·Sabine Müller
Jan 12, 2019·Expert Review of Molecular Diagnostics·Diana GuleiGeorge Adrian Calin
Jul 17, 2019·Nature Biotechnology·Liang QuWensheng Wei
Jan 30, 2020·International Journal of Molecular Sciences·Lewis E FryRobert E MacLaren
May 18, 2020·Current Gene Therapy·Sonali Bhakta, Toshifumi Tsukahara
Jun 25, 2020·International Journal of Molecular Sciences·Pedro MoraisYi-Tao Yu
Dec 3, 2016·Nucleic Acids Research·Jacqueline WettengelThorsten Stafforst
Jan 30, 2019·Nature Biotechnology·Tobias MerkleThorsten Stafforst
Feb 10, 2019·Nature Methods·Dhruva KatrekarPrashant Mali
Oct 29, 2017·Proceedings of the National Academy of Sciences of the United States of America·John R SinnamonGail Mandel
Feb 6, 2020·Nature·Sara Reardon
Dec 29, 2020·Critical Reviews in Biochemistry and Molecular Biology·Emily A ErdmannHeather A Hundley
Dec 26, 2018·Cell Chemical Biology·Leanna R MonteleonePeter A Beal
May 4, 2021·Journal of the American Chemical Society·Erin E DohertyPeter A Beal
Sep 28, 2021·RNA Biology·Hamid Mansouri Khosravi, Michael F Jantsch

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