- Publication describes how the unique qualities of ARCUS nucleases contribute to precise and efficient gene editing for gene insertion, single base editing, specific small and large deletions, and ...

A compact Cas12f nuclease shows high editing efficiency in human cells, with structural insights enabling an engineered variant potentially suited for future AAV‑compatible delivery.

NIH-funded UT team and colleagues from Metagenomi Thereapeutics discovered highly efficient enzyme could enable targeted gene editing within the human body.

The document outlines the non-clinical sequencing and bioinformatics data sponsors need to submit as part of INDs and BLAs for gene-editing therapies.

Dr Annarita Miccio told the ELRIG meeting in March 2026 that gene therapy for sickle cell disease and beta-thalassaemia has entered a more demanding phase, as researchers seek to improve ...