μCas, a novel class of miniature type-V Cas12f nucleases with diverse PAM

Abstract excerpt

Small CRISPR-Cas effectors are key to developing gene editing therapies due to the packaging constraints in viral vectors. While Cas9 and Cas12a CRISPR-Cas effectors have advanced into select clinical applications, their size is prohibitive for efficient delivery of both nuclease and guide RNA in a single viral vector. Type-V Cas12f effectors present a solution given their small size. Here we describe μCas, a nove...

Topics

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• CRISPR and Genetic Engineering
• CRISPR-Associated Proteins
• CRISPR-Cas Systems
• Dependovirus
• Gene Editing
• Genome, Human
• HEK293 Cells
• Mice

Identifiers and source

Literature Corpus work

4150fab1-2676-5737-8cf8-7a09e3e4f87a

DOI

10.1101/2023.02.03.527072