@BroadInstitute a shift from Permanently editing DNA to Temporarily revising RNA – An approach with promise for addressing the risk of developing Alzheimer’s by deactivating APOE4 – RESCUE: RNA Editing for Specific C to U Exchange, the platform builds on REPAIR: RNA Editing for Programmable A to I | Leaders in Pharmaceutical Business Intelligence (LPBI) Group

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@BroadInstitute a shift from Permanently editing DNA to Temporarily revising RNA – An approach with promise for addressing the risk of developing Alzheimer’s by deactivating APOE4 – RESCUE: RNA Editing for Specific C to U Exchange, the platform builds on REPAIR: RNA Editing for Programmable A to I

July 12, 2019 by 2012pharmaceutical

@BroadInstitute a shift from Permanently editing DNA to Temporarily revising RNA – An approach with promise for addressing the risk of developing Alzheimer’s by deactivating APOE4 – RESCUE: RNA Editing for Specific C to U Exchange, the platform builds on REPAIR: RNA Editing for Programmable A to I

Reporter: Aviva Lev-Ari, PhD, RN

2.1.3.14

UPDATED on 8/30/2021

Brief Communication
Published: 30 August 2021

Compact RNA editors with small Cas13 proteins

Nature Biotechnology (2021)Cite this article

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Abstract

CRISPR–Cas13 systems have been developed for precise RNA editing, and can potentially be used therapeutically when temporary changes are desirable or when DNA editing is challenging. We have identified and characterized an ultrasmall family of Cas13b proteins—Cas13bt—that can mediate mammalian transcript knockdown. We have engineered compact variants of REPAIR and RESCUE RNA editors by functionalizing Cas13bt with adenosine and cytosine deaminase domains, and demonstrated packaging of the editors within a single adeno-associated virus.

SOURCE

Kannan, S., Altae-Tran, H., Jin, X. et al. Compact RNA editors with small Cas13 proteins. Nat Biotechnol (2021). https://doi.org/10.1038/s41587-021-01030-2

https://www.nature.com/articles/s41587-021-01030-2

2.1.3.14 @BroadInstitute a shift from Permanently editing DNA to Temporarily revising RNA – An approach with promise for addressing the risk of developing Alzheimer’s by deactivating APOE4 – RESCUE: RNA Editing for Specific C to U Exchange, the platform builds on REPAIR: RNA Editing for Programmable A to I, Volume 2 (Volume Two: Latest in Genomics Methodologies for Therapeutics: Gene Editing, NGS and BioInformatics, Simulations and the Genome Ontology), Part 2: CRISPR for Gene Editing and DNA Repair

The RNA editors converted “the nucleotide base adenine to inosine, or letters A to I. Zhang and colleagues took the REPAIR fusion and evolved it in the lab until it could change cytosine to uridine, or C to U.”
Using Cas13, Zhang’s team was able to take the APOE4 gene — believed to carry the added risk of spurring Alzheimer’s — and changed it to a benign APOE2.

RNA-guided DNA insertion with CRISPR-associated transposases

Jonathan Strecker ¹,²,³,⁴,
Alim Ladha¹,²,³,⁴,
Zachary Gardner¹,²,³,⁴,
Jonathan L. Schmid-Burgk¹,²,³,⁴,
Kira S. Makarova⁵,
Eugene V. Koonin⁵,
Feng Zhang ¹,²,³,⁴,*

¹Broad Institute of MIT and Harvard, Cambridge, MA 02142, USA.
²McGovern Institute for Brain Research, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.
³Department of Brain and Cognitive Sciences, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.
⁴Department of Biological Engineering, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.
⁵National Center for Biotechnology Information, National Library of Medicine, National Institutes of Health, Bethesda, MD 20894, USA.