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Vinogradovas, M.

Publications and source records attributed to Vinogradovas, M..

2 recordsLinked to original sources

Cas12a cleavage and trimming kinetics reveal mismatches as a tool to steer editing

Gene knockouts by CRISPR-Cas nucleases rely on targeted DNA cleavage and error-prone DNA repair: end-joining pathways can introduce insertions and deletions that assist in disrupting the coding sequence. However, only a fraction of edits achieves this, and an unfavorable array of repair outcomes typically requires switching to another editing technology. Key factors that influence repair are the types and lengths of DNA ends following cleavage. Here, we investigated Cas12a's ability to produce different ends and if they can be used to redistribute editing outcomes. We determined the sites and rates of target cleavage by Cas12a in vitro by combining kinetic modeling with nucleotide-resolution assays. Here, we show that trimming - repeated cleavage of an already cut target - occurs about 4x faster than initial cleavage; it also presents alternative DNA end structures for cellular repair. We also introduced specific mismatches to the gRNA: Cas12a maintained fast target cleavage, but changed where the target was cleaved and how quickly it was trimmed, compared to matched gRNA. We exploited the differences in cleavage kinetics between matched and mismatched gRNAs to develop reprogrammed gRNAs, i.e. rpgRNAs. Intentionally-mismatched rpgRNAs retained the high editing efficiency observed with traditional gRNAs. However, they shifted editing between in-frame and out-of-frame outcomes to enhance gene knockout success across genes. Reprogrammed gRNAs offer an efficient way to steer editing toward such preferred outcomes, while retaining the simplicity of gene editing with CRISPR-Cas nucleases.

biochemistry↗

Supercoiling twists Cas9 off-target discrimination when nicking and cleaving

Programmed with an RNA guide, Cas9 nuclease directs double-strand DNA cleavage via its two nuclease domains. However, Cas9 sometimes falsely identifies DNA targets by binding and cleaving DNA that does not match its PAM and guide RNA sequence requirements. Cas9s specificity is often affected by DNA topology, as DNA negative supercoiling can increase off-target activity while positive supercoiling can even prevent on-target activity. Such dramatic DNA topological changes routinely occur in cells as a result of transcription and replication, making Cas9s specificity a challenge for gene editing. To determine how Cas9 imparts its specificity across sequences and topologies, we directly mapped kinetic cleavage rates and sites for thousands of relaxed and negatively-supercoiled target sequences via NucleaSeq. We find that: Negative supercoiling can accelerate off-target cleavage a thousand-fold, and shift cleavage sites by two nucleotides. Guide-target mispairs differently impact RuvC and HNH domains, which can lead to topology-dependent nicking by Cas9. Finally, we predict these variations in Cas9 cleavage activity with a biophysical model that accounts for DNA topological state. These efforts expose Cas9s strand-specific off-target cleavage activity and can improve off-target identification for more predictable and safer gene editing. O_FIG O_LINKSMALLFIG WIDTH=200 HEIGHT=94 SRC="FIGDIR/small/715559v1_ufig1.gif" ALT="Figure 1"> View larger version (20K): org.highwire.dtl.DTLVardef@1c1d4c5org.highwire.dtl.DTLVardef@77d891org.highwire.dtl.DTLVardef@2b3073org.highwire.dtl.DTLVardef@179d442_HPS_FORMAT_FIGEXP M_FIG C_FIG

biochemistry↗