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Programmable RNA editing by recruiting endogenous ADAR using engineered RNAs.

Abstract：

:Current tools for targeted RNA editing rely on the delivery of exogenous proteins or chemically modified guide RNAs, which may lead to aberrant effector activity, delivery barrier or immunogenicity. Here, we present an approach, called leveraging endogenous ADAR for programmable editing of RNA (LEAPER), that employs short engineered ADAR-recruiting RNAs (arRNAs) to recruit native ADAR1 or ADAR2 enzymes to change a specific adenosine to inosine. We show that arRNA, delivered by a plasmid or viral vector or as a synthetic oligonucleotide, achieves editing efficiencies of up to 80%. LEAPER is highly specific, with rare global off-targets and limited editing of non-target adenosines in the target region. It is active in a broad spectrum of cell types, including multiple human primary cell types, and can restore α-L-iduronidase catalytic activity in Hurler syndrome patient-derived primary fibroblasts without evoking innate immune responses. As a single-molecule system, LEAPER enables precise, efficient RNA editing with broad applicability for therapy and basic research.

journal_name

Nat Biotechnol

journal_title

Nature biotechnology

authors

Qu L,Yi Z,Zhu S,Wang C,Cao Z,Zhou Z,Yuan P,Yu Y,Tian F,Liu Z,Bao Y,Zhao Y,Wei W

doi

10.1038/s41587-019-0178-z

subject

Has Abstract

pub_date

2019-09-01 00:00:00

pages

1059-1069

issue

9

eissn

1087-0156

issn

1546-1696

pii

10.1038/s41587-019-0178-z

journal_volume

37

pub_type

杂志文章

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