RNA editing with CRISPR-Cas13
Broad Institute · McGovern Institute for Brain Research · +5 more institutions
Abstract
Nucleic acid editing holds promise for treating genetic disease, particularly at the RNA level, where disease-relevant sequences can be rescued to yield functional protein products. Type VI CRISPR-Cas systems contain the programmable single-effector RNA-guided ribonuclease Cas13. We profiled type VI systems in order to engineer a Cas13 ortholog capable of robust knockdown and demonstrated RNA editing by using catalytically inactive Cas13 (dCas13) to direct adenosine-to-inosine deaminase activity by ADAR2 (adenosine deaminase acting on RNA type 2) to transcripts in mammalian cells. This system, referred to as RNA Editing for Programmable A to I Replacement (REPAIR), which has no strict sequence constraints, can…
Citation impact
- FWCI
- 58.87
- Percentile
- 100%
- References
- 37
Authors
7- DCDavid CoxCorresponding
Broad Institute, McGovern Institute for Brain Research, Harvard–MIT Division of Health Sciences and Technology, IIT@MIT, Massachusetts Institute of Technology
- JSJonathan S. GootenbergCorresponding
Broad Institute, Harvard University, McGovern Institute for Brain Research, IIT@MIT, Center for Systems Biology, Massachusetts Institute of Technology
- OOOmar O. AbudayyehCorresponding
Broad Institute, McGovern Institute for Brain Research, Harvard–MIT Division of Health Sciences and Technology, IIT@MIT, Massachusetts Institute of Technology
- BFBrian Franklin
Broad Institute, McGovern Institute for Brain Research, IIT@MIT, Massachusetts Institute of Technology
- MJMax J. Kellner
Broad Institute, McGovern Institute for Brain Research, IIT@MIT, Massachusetts Institute of Technology
Topics & keywords
- RNA editing
- CRISPR
- RNA
- Genome editing
- Biology
- Computational biology
- ADAR
- Gene knockdown
Funding
- HHHoward Hughes Medical InstituteAward: award352313
- SFSimons FoundationAward: award352312
- PGPaul G. Allen Family FoundationAward: award352315
- NYNew York Stem Cell FoundationAward: award352314
- PAPaul and Daisy Soros Fellowships for New AmericansAward: award352310
- VFVallee FoundationAward: award352316
- NINational Institutes of HealthAwards: award352307, 1F30-CA210382, 1R01-MH110049, 1R01-HG009761, award352311, award352308, 1DP1-HL141201, award352317, award352309, T32GM007753