Compact RNA editors with small Cas13 proteins

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 trans...

Full description

Bibliographic Details
Main Authors: Kannan, Soumya, Altae-Tran, Han, Jin, Xin, Madigan, Victoria J, Oshiro, Rachel, Makarova, Kira S, Koonin, Eugene V, Zhang, Feng
Other Authors: Howard Hughes Medical Institute
Format: Article
Language:English
Published: Springer Science and Business Media LLC 2022
Online Access:https://hdl.handle.net/1721.1/144412
Description
Summary: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.