CRISPR-Cas9 genome editing articles from across Nature Portfolio

Quantifying genomic aberrations resulting from designer nucleases activity is essential for gene therapy clinical translation. Here, the authors present a modular digital PCR technique that profiles DNA repair precision and cut-repair cycles at the edited loci, exposing current evaluation biases.

Current sterile insect approaches require labor-intensive sorting. Here, authors develop a temperature-controlled Cas12a system in Drosophila that produces sterile males and eliminates females, offering a scalable genetic tool for controlling pests and disease vectors.

Cut-seq1, Cut-seq2, DeepCut and CLOVE-seq methods find optimized single-guide RNAs to detect a large number of rare variants in cells.

Hybrid gRNAs significantly increase targeted editing in the liver while simultaneously reducing unwanted bystander editing in humanized mouse models.

CRISPR base editors directly modify the non-target strand (NTS). Here, authors reveal an activity by miniature Cas12f cytosine base editor (CBE) to also edit the TS, and next engineer a TS-preferential CBE. The work establishes a strand-selectable miniature CBE toolkit with therapeutic potential.

Progress in malaria control has stalled in recent years. Here, authors present a self-limiting gene drive, Male Drive Female Sterile (MDFS), which spreads dominant female sterility via fertile males and can eliminate mosquito populations in the lab, offering a promising tool for vector suppression.

Growth rings in fossilized bones suggest the diminutive dinosaur, Nanotyrannus, would never grow up to become a T. rex — plus, a small breast cancer prevention trial shows promise.

In a world first, a bespoke gene-editing therapy benefited one child. Now researchers plan to launch a clinical trial of the approach.

CRISPR-based tools can’t easily access the DNA in these organelles, but researchers are finding other ways in.

CRISPR screens are widely used to identify essential genes and potential drug targets, but typical setups were designed for the study of highly proliferative cancer cell lines. Now, a protocol, using inducible Cas9, enables screens of non-proliferative cell states including senescence, quiescence and terminal differentiation, thereby expanding the applicability of CRISPR screens.

Genome editors are molecular machines that can rewrite the genetic code in cells, but sometimes they produce errors in the form of unintended sequence insertions or deletions, collectively known as indel errors. Genome editors have now been engineered that make up to 60-fold fewer indel errors than previous ones did.

We developed a customized base editing strategy to efficiently, precisely and safely correct the most common ACTA2 pathogenic mutation in multisystemic smooth muscle dysfunction syndrome. In vivo delivery of the bespoke base editor prolongs survival and rescues systemic phenotypes in a mouse model of multisystemic smooth muscle dysfunction syndrome.