Compact RNA editors with small Cas13 proteins

被引：104

作者：

Kannan, Soumya ^{[1
,2
,3
,4
,5
]}

Altae-Tran, Han ^{[1
,2
,3
,4
,5
]}

Jin, Xin ^{[1
,2
,3
,4
,5
,6
,7
]}

Madigan, Victoria J. ^{[1
,2
,3
,4
,5
]}

Oshiro, Rachel ^{[1
,2
,3
,4
,5
]}

Makarova, Kira S. ^{[8
]}

Koonin, Eugene, V ^{[8
]}

Zhang, Feng ^{[1
,2
,3
,4
,5
]}

机构：

[1] Howard Hughes Med Inst, Cambridge, MA 02138 USA

[2] Broad Inst MIT & Harvard, Cambridge, MA 02142 USA

[3] MIT, McGovern Inst Brain Res MIT, 77 Massachusetts Ave, Cambridge, MA 02139 USA

[4] MIT, Dept Biol Engn, 77 Massachusetts Ave, Cambridge, MA 02139 USA

[5] MIT, Dept Brain & Cognit Sci, E25-618, Cambridge, MA 02139 USA

[6] Harvard Univ, Cambridge, MA 02138 USA

[7] Harvard Univ, Dept Stem Cell & Regenerat Biol, Cambridge, MA 02138 USA

[8] NLM, Natl Ctr Biotechnol Informat, NIH, Bethesda, MD USA

来源：

NATURE BIOTECHNOLOGY | 2022年 / 40卷 / 02期

基金：

美国国家科学基金会;

关键词：

SYSTEM;

D O I：

10.1038/s41587-021-01030-2

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

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.

引用

页码：194 / +

页数：9

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