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Leber hereditary optic neuropathy (LHON) is caused by mtDNA mutations. Here, authors generated a mouse model with the common LHON mutation and showed that optimized TALE-like deaminases restored both phenotype and genotype via high-fidelity mitochondrial base editing.

Cpf1 is a type V CRISPR effector protein that has different target and guide RNA requirements compared to Cas9, thus offering an addition tool for precision genome editing. Here Kimet al. show that Cpf1 ribonucleoprotein can be introduced into protoplasts and used for transgene-free gene editing in plants.

Cas9 RNA-guided engineered nucleases (RGENs) induce site-specific DNA cleavages in cultured cells and organisms and are used widely as genome-editing tools. Here, the authors develop an RGEN-based technology to genotype both RGEN-induced mutations and cancer-associated mutations in human cell lines.

Nature Biotechnology asks selected members of the international community to comment on the ethical issues raised by the prospect of CRISPR-Cas9 engineering of the human germline.

Nature Biotechnology asks a selection of researchers about the most exciting frontier in their field and the most needed technologies for advancing knowledge and applications.

Cas12a is a RNA-guided DNA endonuclease whose detailed mechanisms of target searching and DNA cleavage remained unclear. Here authors use single-molecule fluorescence assays to show that Cas12a searches for their on-target site.

Cas9 and Cpf1 have both been adapted for genome engineering, editing and gene expression regulation. Here the authors design a fusion guide RNA that can interact with both proteins for multiple and orthogonal genome manipulation.

Split DddA-derived base editors fused to TALEs enable mitochondrial DNA editing. Here the authors demonstrate their use in mouse embryos with germline transmission.

To celebrate the first 20 years of Nature Reviews Genetics, we asked 12 leading scientists to reflect on the key challenges and opportunities faced by the field of genetics and genomics.

Anomalous Arctic warming has been linked to colder North American winters. Analyses of weather and productivity observations reveal that Arctic–North American teleconnections reduce gross primary productivity in the US.

Kim et al. used directed evolution methods to identify a high-fidelity SpCas9 variant, Sniper2L, which exhibits high general activity but maintains high specificity at a large number of target sites.

Cas9 nickases (nCas9s) produce nicks or single-strand breaks in the DNA. Here the authors analyse the on- and off-target nicks generated by these nickases, and show that nCas9 (H840A) but not nCas9 (D10A) can cleave both strands and produce unwanted DNA double-strand breaks.

This study shows that increased fire risk under global warming may not decrease in line with CO₂ reductions. The exacerbated fire danger is projected to contribute to extra CO₂ emissions in 68% of the world due to the hysteresis of climate responses to CO₂ levels.

The amount of genetic material that can be packaged in AAV vectors used for genome editing is limited. Here the authors show that the smallest known Cas9 orthologue, cjCas9, can be packaged in a single AAV vector along with sgRNA and a marker gene, and demonstrate efficient gene editing in mice.

Uracil DNA glycosylase-fused TALE-linked deaminases achieve precision A-to-G base editing without bystander C-to-T editing in chloroplast DNA to create herbicide-resistant plants with a heritable homoplasmic mutation in the psbA gene.

CRISPR–Cas9 has truly democratized genome editing. In this Perspective, Jin-Soo Kim discusses CRISPR–Cas9 genome editing in the context of earlier innovations using meganucleases, ZFNs and TALENs, which paved the way for the ongoing CRISPR–Cas revolution.

On-target and off-target specificities of Cfp1 are assessed and compared with SpCas9 in human cells.

Mice with targeted point mutations are generated efficiently using Cas9–cytidine deaminase fusions.

CRISPR–Cas9 genome editing is used to induce a DNA repair response and correct a disease-causing heterozygous mutation in human embryos with reduced mosaicism and preferential repair using the wild-type copy of the gene.

Adenine base editing is used to treat a mouse model of Duchenne muscular dystrophy and to create defined mutations in mouse embryos.

The genome of human cells is edited using the bacterial RNA-guided Cas9 endonuclease.

Base editors composed of Cas9 fused to a deaminase show high specificity in genome-wide analyses.

Attempts to image activated macrophages in vivo have been hampered by selectivity and delivery problems. Here the authors develop a small molecule fluorescent probe specific to activated M1 and M2 macrophages, identify the orphan receptor Slc18b1/SLC18B1 as the mechanism of uptake, and use it to image atherosclerosis in mice.

CRISPR-Cas9 genome editing is achieved in Arabidopsis, tobacco, lettuce and rice without introducing foreign DNA into the plants.

This study showed efficient base editing of mitochondria and chloroplast genomes in both lettuce and rapeseed by DddA-derived cytosine base editors (DdCBEs), and DNA-free editing in chloroplasts by delivering DdCBE mRNA to protoplasts, which avoids off-target effects.

The CRISPR endonuclease LbCpf1 is reported to have greater efficiency and specificity than Cas9. Here, the authors use LbCpf1 to target the angiogenesis-related genes VEGF and HIF1a, and show that delivery of the nuclease using AAV9 is effective in mouse models of macular degeneration.

Cas9 can induce extensive on-target damage, including large deletions, inversions, and insertions.

In addition to causing A-to-G base transitions, adenine base editors also cause C base substitutions

Cas12a-linked base editors can broaden the targeting scope of programmable cytidine deaminases. Here the authors assess their target specificity in an in vitro genome-wide assay.

A simple episomal fluorescent reporter for flow cytometric enrichment of cells with zinc-finger nuclease– or TALE nuclease–induced genomic modifications is described.

An international team of researchers finds high potential for improving climate projections by a more comprehensive treatment of largely ignored Arctic vegetation types, underscoring the importance of Arctic energy exchange measuring stations.

Plant-compatible adenine base editor systems are now demonstrated to work in protoplasts and individual plants of Arabidopsis thaliana and Brassica napus, yielding plants with predicted nucleotide substitutions and stably inherited phenotypes.

In vitro digestion of genomic DNA with Cas9 and single guide RNAs (sgRNAs) yields genome-wide off-target sites at frequencies below 0.1%. Off-target sites can be further reduced with modified sgRNAs.

Plant-optimized transcription activator-like effector-linked deaminases enable site-specific A-to-G base editing in the chloroplast genome, leading to heritable homoplasmic base conversions and phenotypic changes.

The development, characterization and phase I clinical testing of the RAF inhibitor belvarafenib in cancer and a new resistance mechanism mediated by ARAF mutations are described.