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

Publication ,  Journal Article
Katrekar, D; Chen, G; Meluzzi, D; Ganesh, A; Worlikar, A; Shih, Y-R; Varghese, S; Mali, P
Published in: Nat Methods
March 2019

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.

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Published In

Nat Methods

DOI

EISSN

1548-7105

Publication Date

March 2019

Volume

16

Issue

3

Start / End Page

239 / 242

Location

United States

Related Subject Headings

  • RNA, Messenger
  • RNA, Guide, CRISPR-Cas Systems
  • RNA Splicing
  • RNA Editing
  • Point Mutation
  • Mice
  • Humans
  • High-Throughput Nucleotide Sequencing
  • HEK293 Cells
  • Disease Models, Animal
 

Citation

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Katrekar, D., Chen, G., Meluzzi, D., Ganesh, A., Worlikar, A., Shih, Y.-R., … Mali, P. (2019). In vivo RNA editing of point mutations via RNA-guided adenosine deaminases. Nat Methods, 16(3), 239–242. https://doi.org/10.1038/s41592-019-0323-0
Katrekar, Dhruva, Genghao Chen, Dario Meluzzi, Ashwin Ganesh, Atharv Worlikar, Yu-Ru Shih, Shyni Varghese, and Prashant Mali. “In vivo RNA editing of point mutations via RNA-guided adenosine deaminases.Nat Methods 16, no. 3 (March 2019): 239–42. https://doi.org/10.1038/s41592-019-0323-0.
Katrekar D, Chen G, Meluzzi D, Ganesh A, Worlikar A, Shih Y-R, et al. In vivo RNA editing of point mutations via RNA-guided adenosine deaminases. Nat Methods. 2019 Mar;16(3):239–42.
Katrekar, Dhruva, et al. “In vivo RNA editing of point mutations via RNA-guided adenosine deaminases.Nat Methods, vol. 16, no. 3, Mar. 2019, pp. 239–42. Pubmed, doi:10.1038/s41592-019-0323-0.
Katrekar D, Chen G, Meluzzi D, Ganesh A, Worlikar A, Shih Y-R, Varghese S, Mali P. In vivo RNA editing of point mutations via RNA-guided adenosine deaminases. Nat Methods. 2019 Mar;16(3):239–242.

Published In

Nat Methods

DOI

EISSN

1548-7105

Publication Date

March 2019

Volume

16

Issue

3

Start / End Page

239 / 242

Location

United States

Related Subject Headings

  • RNA, Messenger
  • RNA, Guide, CRISPR-Cas Systems
  • RNA Splicing
  • RNA Editing
  • Point Mutation
  • Mice
  • Humans
  • High-Throughput Nucleotide Sequencing
  • HEK293 Cells
  • Disease Models, Animal