Mike Firth

TL;DR: VIVO provides a general strategy for defining and quantifying the off-target effects of gene-editing nucleases in whole organisms, thereby providing a blueprint to foster the development of therapeutic strategies that use in vivo gene editing.

TL;DR: Combining lactate transport inhibition by AZD3965 with GLS1 inhibition in vitro, enhanced cell growth inhibition and cell death compared to monotherapy treatment and the ability to combine AZD 3965 with novel, and standard-of-care inhibitors offers novel combination opportunities in haematological cancers.

TL;DR: The authors present CRISPR GUARD, which uses very short non-cleaving gRNAs to prevent editing at off-target sites, and discover that short guide RNAs can also support base editing if they contain cytosines within the deaminase activity window.

TL;DR: A Base Editing FLuorescent Activity REporter (BE-FLARE), which allows for the enrichment of cells that have undergone editing of target loci based on a fluorescence shift from BFP to GFP, is developed and workflows for the accelerated generation of genetically engineered cell models and the discovery of alternative base editors are outlined.

TL;DR: In vitro studies on synovial fibroblasts obtained from healthy wild-type, arthritic Tg 197, and infliximab-treated Tg197 transgenic mice confirmed the association of itaconic acid with rheumatoid arthritis and disease-moderating drug effects.