In vivo RNA editing of point mutations via RNA-guided adenosine deaminases

We utilized a system for sequence-specific RNA base editing via Adenosine Deaminases acting on RNA (ADAR) enzymes with associated ADAR guide RNAs (adRNAs). We systematically engineered it to harness ADARs, and comprehensively evaluated its specificity and activity in vitro and in vivo via two mouse...

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Autores principales: Katrekar, Dhruva, Chen, Genghao, Meluzzi, Dario, Ganesh, Ashwin, Worlikar, Atharv, Shih, Yu-Ru, Varghese, Shyni, Mali, Prashant
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2019
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6395520/
https://www.ncbi.nlm.nih.gov/pubmed/30737497
http://dx.doi.org/10.1038/s41592-019-0323-0
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author Katrekar, Dhruva
Chen, Genghao
Meluzzi, Dario
Ganesh, Ashwin
Worlikar, Atharv
Shih, Yu-Ru
Varghese, Shyni
Mali, Prashant
author_facet Katrekar, Dhruva
Chen, Genghao
Meluzzi, Dario
Ganesh, Ashwin
Worlikar, Atharv
Shih, Yu-Ru
Varghese, Shyni
Mali, Prashant
author_sort Katrekar, Dhruva
collection PubMed
description We utilized a system for sequence-specific RNA base editing via Adenosine Deaminases acting on RNA (ADAR) enzymes with associated ADAR guide RNAs (adRNAs). We systematically engineered it to harness ADARs, and comprehensively evaluated its specificity and activity in vitro and in vivo via two mouse models of human disease. We anticipate this platform will enable tunable and reversible engineering of RNAs for diverse applications.
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spelling pubmed-63955202019-08-08 In vivo RNA editing of point mutations via RNA-guided adenosine deaminases Katrekar, Dhruva Chen, Genghao Meluzzi, Dario Ganesh, Ashwin Worlikar, Atharv Shih, Yu-Ru Varghese, Shyni Mali, Prashant Nat Methods Article We utilized a system for sequence-specific RNA base editing via Adenosine Deaminases acting on RNA (ADAR) enzymes with associated ADAR guide RNAs (adRNAs). We systematically engineered it to harness ADARs, and comprehensively evaluated its specificity and activity in vitro and in vivo via two mouse models of human disease. We anticipate this platform will enable tunable and reversible engineering of RNAs for diverse applications. 2019-02-08 2019-03 /pmc/articles/PMC6395520/ /pubmed/30737497 http://dx.doi.org/10.1038/s41592-019-0323-0 Text en Users may view, print, copy, and download text and data-mine the content in such documents, for the purposes of academic research, subject always to the full Conditions of use:http://www.nature.com/authors/editorial_policies/license.html#terms
spellingShingle Article
Katrekar, Dhruva
Chen, Genghao
Meluzzi, Dario
Ganesh, Ashwin
Worlikar, Atharv
Shih, Yu-Ru
Varghese, Shyni
Mali, Prashant
In vivo RNA editing of point mutations via RNA-guided adenosine deaminases
title In vivo RNA editing of point mutations via RNA-guided adenosine deaminases
title_full In vivo RNA editing of point mutations via RNA-guided adenosine deaminases
title_fullStr In vivo RNA editing of point mutations via RNA-guided adenosine deaminases
title_full_unstemmed In vivo RNA editing of point mutations via RNA-guided adenosine deaminases
title_short In vivo RNA editing of point mutations via RNA-guided adenosine deaminases
title_sort in vivo rna editing of point mutations via rna-guided adenosine deaminases
topic Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6395520/
https://www.ncbi.nlm.nih.gov/pubmed/30737497
http://dx.doi.org/10.1038/s41592-019-0323-0
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