RNA editing

Biology

Gene

Gene knockdown

Cytosine

Computational biology

Cell biology

CRISPR

Genetics

Nature Biotechnology

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. Compact Cas13 proteins are used to design RNA editors that fit into a single adeno-associated virus.

Compact RNA editors with small Cas13 proteins