Compact RNA editors with small Cas13 proteins

带有小型 Cas13 蛋白的紧凑型 RNA 编辑器

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作者:Soumya Kannan #, Han Altae-Tran #, Xin Jin, Victoria J Madigan, Rachel Oshiro, Kira S Makarova, Eugene V Koonin, Feng Zhang3

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.

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