ioCRISPR-Ready Cells have been built for scientists looking to generate high-efficiency gene knockouts in physiologically relevant human cells. Based on our opti-ox™ powered ioWild Type Cells™, these cells offer users a well-defined and characterised human model with high lot-to-lot consistency and simple protocols for handling and culturing. Each model can be used with an optimised protocol for guide RNA (gRNA) delivery that helps maximise knockout efficiency. Scale from single gene knockouts to pooled or arrayed CRISPR screens for applications including functional genomics, disease model generation, drug target identification and fundamental human biology research.
ioCells™ are for research use only.
As ioCRISPR-Ready Cells are powered by opti-ox precision cell reprogramming, they have consistency and scalability built-in. With these cells, users can significantly cut experimental timelines by no longer needing to spend months engineering and characterising their own Cas9 stable iPSC lines or optimising differentiation protocols.
“Using the conventional system of directed differentiation followed by transduction of Cas9 and gRNAs, we could only do about one genome-level screen per year. With ioCRISPR-Ready Cells, we are now able to do 6-7 of these screens per year. The model almost works too well because now we have all this data to process, analyse, and publish”
Emmanouil Metzakopian, PhD
VP of Research and Development, bit.bio
Researchers ran a genome-wide knockout screen in opti-ox powered glutamatergic neurons constitutively expressing Cas9 nuclease with the goal of identifying genes that regulate a cold shock protein known as RBM3, a gene associated with brain recovery after damage that is typically switched off. The researchers identified a set of splicing factors highly associated with RBM3 expression, identifying a pathway that may become a therapeutic target in the treatment of neurodegenerative conditions.
Using opti-ox powered glutamatergic neurons constitutively expressing Cas9, researchers ran a targeted knockout screen on 4401 druggable proteins specifically looking to identify genes involved in the unfolded protein response (UPR). This pathway causes cell death in response to excessive protein aggregation, often observed in neurodegenerative proteinopathies. A follow-up arrayed knockout screen of 188 hits yielded 13 targets that were subject to further assays, leading them to a single target KAT2B. KAT2B inhibition with L-moses led to a significant reduction in UPR-mediated cell death, offering a potential therapeutic modality for proteinopathies like Alzheimer’s disease.