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


Journal

Nature methods
ISSN: 1548-7105
Titre abrégé: Nat Methods
Pays: United States
ID NLM: 101215604

Informations de publication

Date de publication:
03 2019
Historique:
received: 16 08 2018
accepted: 13 12 2018
pubmed: 10 2 2019
medline: 23 5 2019
entrez: 10 2 2019
Statut: ppublish

Résumé

We present in vivo sequence-specific RNA base editing via adenosine deaminases acting on RNA (ADAR) enzymes with associated ADAR guide RNAs (adRNAs). To achieve this, we systematically engineered adRNAs to harness ADARs, and comprehensively evaluated the specificity and activity of the toolsets in vitro and in vivo via two mouse models of human disease. We anticipate that this platform will enable tunable and reversible engineering of cellular RNAs for diverse applications.

Identifiants

pubmed: 30737497
doi: 10.1038/s41592-019-0323-0
pii: 10.1038/s41592-019-0323-0
pmc: PMC6395520
mid: NIHMS1516811
doi:

Substances chimiques

RNA, Guide 0
RNA, Messenger 0
Adenosine Deaminase EC 3.5.4.4

Types de publication

Journal Article Research Support, N.I.H., Extramural

Langues

eng

Sous-ensembles de citation

IM

Pagination

239-242

Subventions

Organisme : NIGMS NIH HHS
ID : R01 GM123313
Pays : United States
Organisme : NCI NIH HHS
ID : R01 CA222826
Pays : United States
Organisme : NIDDK NIH HHS
ID : K01 DK119687
Pays : United States
Organisme : NHGRI NIH HHS
ID : R01 HG009285
Pays : United States
Organisme : NIDDK NIH HHS
ID : F32 DK112682
Pays : United States

Commentaires et corrections

Type : CommentIn
Type : CommentIn

Références

Nature. 1996 Feb 1;379(6564):460-4
pubmed: 8559253
Science. 2016 Jan 22;351(6271):400-3
pubmed: 26721683
Hum Mutat. 2008 Aug;29(8):1037-47
pubmed: 18454449
Science. 2016 Jan 22;351(6271):407-411
pubmed: 26721686
Nat Biotechnol. 2016 Mar;34(3):334-8
pubmed: 26829317
Nat Methods. 2018 Jul;15(7):535-538
pubmed: 29967493
Proc Natl Acad Sci U S A. 2013 Nov 5;110(45):18285-90
pubmed: 24108353
J Cell Sci. 2003 May 1;116(Pt 9):1805-18
pubmed: 12665561
Science. 2016 Jan 22;351(6271):403-7
pubmed: 26721684
Nucleic Acids Res. 2017 Mar 17;45(5):2797-2808
pubmed: 27907896
Gene Ther. 2017 Dec;24(12):779-786
pubmed: 28984845
Genome Biol. 2017 Oct 23;18(1):195
pubmed: 29061182
Proc Natl Acad Sci U S A. 1995 Aug 29;92(18):8298-302
pubmed: 7545300
RNA Biol. 2018 Jan 2;15(1):104-114
pubmed: 29099293
Science. 2017 Nov 24;358(6366):1019-1027
pubmed: 29070703
Sci Rep. 2017 Feb 02;7:41478
pubmed: 28148949
Proc Natl Acad Sci U S A. 2017 Oct 31;114(44):E9395-E9402
pubmed: 29078406
Proc Natl Acad Sci U S A. 1989 Jun;86(11):4142-6
pubmed: 2471197
Cell. 2018 Feb 8;172(4):811-824.e14
pubmed: 29395325
Nat Med. 2013 Feb;19(2):209-16
pubmed: 23291631

Auteurs

Dhruva Katrekar (D)

Department of Bioengineering, University of California, San Diego, CA, USA.

Genghao Chen (G)

Department of Bioengineering, University of California, San Diego, CA, USA.

Dario Meluzzi (D)

Department of Bioengineering, University of California, San Diego, CA, USA.

Ashwin Ganesh (A)

Department of Bioengineering, University of California, San Diego, CA, USA.

Atharv Worlikar (A)

Department of Bioengineering, University of California, San Diego, CA, USA.

Yu-Ru Shih (YR)

Department of Bioengineering, University of California, San Diego, CA, USA.
Department of Biomedical Engineering, Duke University, Durham, NC, USA.

Shyni Varghese (S)

Department of Bioengineering, University of California, San Diego, CA, USA.
Department of Biomedical Engineering, Duke University, Durham, NC, USA.

Prashant Mali (P)

Department of Bioengineering, University of California, San Diego, CA, USA. pmali@ucsd.edu.

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Classifications MeSH