Running Large-scale CRISPR Screens in Human Neurons
This webinar will showcase two peer-reviewed studies using opti-ox™ powered human neurons constitutively expressing Cas9 for the generation of large scale CRISPR screens for the identification of new therapeutic targets for the treatment of neurodegenerative diseases.
This webinar will showcase two peer-reviewed studies using opti-ox™ powered human neurons constitutively expressing Cas9 for the generation of large scale CRISPR screens for the identification of new therapeutic targets for the treatment of neurodegenerative diseases.
Identifying therapeutic targets for neurodegenerative conditions is often challenging due to the limited accessibility of reproducible, scalable in vitro cell models. Genome-level CRISPR screens are useful for these studies but performing screens that include the necessary replicates requires billions of cells. Human iPSC-derived cells can provide the needed scale, however, the complex process of directed differentiation is time-consuming, resource-intensive, and rarely feasible. Furthermore, delivering ribonucleases by transfection or transduction is inefficient in human iPSC-derived cells, especially delicate cell types like neurons. As a result, scientists often rely on immortalised cell lines, which do not accurately represent human biology or disease states, to run large-scale CRISPR screens.
In this GEN webinar, two experts will discuss solutions for running large-scale CRISPR screens to identify therapeutic targets for neurodegenerative diseases. They will present ioCRISPR-Ready Cells™: human iPSC-derived cells precision reprogrammed with opti-ox™, that constitutively express Cas9 nuclease, which are built for rapidly generating gene knockouts and CRISPR screens. During the webinar, you’ll learn about two peer-reviewed studies that performed large scale CRISPR knockout screens using opti-ox powered glutamatergic neurons with stable Cas9 expression. The first study demonstrates a loss of function genetic screen using a human druggable genome library. The second study investigated possible regulators of the RNA binding motif 3 protein, whose enhanced expression is highly neuroprotective both in vitro and in vivo.
In this webinar, you will learn about:
- ioCRISPR-Ready Cells: development and applications.
- The use of these cells in a large-scale loss-of-function knockout screen that identified 13 druggable targets for protection against cell death pathways observed in neurodegenerative proteinopathies.
- A genome-wide knockout screen in these cells that elucidated how neuroprotective protein RMB3 expression is regulated, identifying a new potential therapeutic for neurodegenerative disease.