CRISPR-Select Validates Drug Targets and Drug Responders Early and Conclusively | Wednesday June 28, 2023 | 3:00 pm–4:00 pm CEST / 9:00 am–10:00 am EDT
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Taking the Guesswork Out of Drug Development: CRISPR-Select Validates Drug Targets and Drug Responders Early and Conclusively
A variety of CRISPR-based approaches are routinely used at all critical steps of the drug development process, e.g., target validation and drug responder prediction. However, despite their widespread use, all of the currently-used approaches lack watertight controls to allow conclusive results. Furthermore, the current approaches are typically non-quantitative, have proliferation/survival as the sole cellular readout, or are costly and time-consuming, e.g., when based on the generation of clonal cell lines.
To address these limitations, scientists at Biotech Research & Innovation Centre, University of Copenhagen, have developed CRISPR-Select, a conclusive functional genetic assay that is also quantitative, simple, cost-efficient and fast, and compatible with any physiological or pathological cellular readout for which a FACS marker exists. CRISPR-Select, which was recently described in an article published in Nature Genetics, tracks and quantitates the genome editing event-of-interest relative to a built-in neutral control mutation in the edited cell population as a function of either time, space or cell state, as measured by FACS.
This versatile functional assay can conclusively address all critical questions in the drug and drug target development process, including:
- Is my protein or gene a good therapeutic target?
- How should a drug best block my target (e.g. catalytic inhibition, protein-protein interaction inhibition, elimination of expression)?
- Does my drug act on the intended target?
- Is a given genetic variant/mutation benign or pathogenic?
- Which patient genotypes will respond to my drug?
What will you learn from this webinar:
The principles of CRISPR-Select
Design of functional genetic assays based on CRISPR-Select
Uses of CRISPR-Select in the drug and drug target development pipeline
- 15.00 Welcome and introduction by CRISPR Medicine News
- 15.05 Morten Frödin PhD |CRISPR-Select Validates Drug Targets and Drug Responders Early and Conclusively
- 15.45 Q & A with Morten Frödin PhD
- 16.00 Close by CRISPR Medicine News
Speaker | Title:
Morten Frödin PhD,
Associate Professor, Group Leader at Biotech Research & Innovation Centre (BRIC), University of Copenhagen & Co-founder of BioPhenyx | CRISPR-Select Validates Drug Targets and Drug Responders Early and Conclusively
Morten Frödin PhD, Associate Professor, Group Leader at Biotech Research & Innovation Centre (BRIC), University of Copenhagen
Dr. Morten Frödin received his PhD in cell biology from the University of Copenhagen (Denmark) in 1997. He subsequently spent one year working as a research fellow within gene targeting in mice at the Pasteur Institute, Paris (France). Since 2004, he has been an Associate Professor and Group Leader at Biotech Research and Innovation Centre, University of Copenhagen. His research has focused on growth factor signal transduction via protein kinase cascades, and has increasingly moved towards the generation and study of genetic mouse and cell models of disease. His lab has invested considerable efforts in developing new genome-editing technologies. Dr. Frödin pioneered the use of single-stranded oligonucleotides (ssODNs) as knockin donors in collaboration with Sigma scientists (Chen et al., 2011, Nature Methods, 8:753-755), FACS-based genome editing (Duda et al., 2014, Nucleic Acids Research, 42:e84; Lonowski et al., 2017, Nature Protocols, 12:581-603), in vivo engineering of oncogenes in the adult mouse liver (Engelholm et al., 2017, Gastroenterology, 153:1662-1673) and most recently, CRISPR-Select functional genetic analysis (Niu et al., 2022, Nature Genetics, 54:1983-1993). CRISPR-Select was developed in collaboration with Professor Claus Storgaard Sørensen, Biotech Research and Innovation Centre, University of Copenhagen, with whom he has also founded the company BioPhenyx to serve pharma/biotech using CRISPR-Select.
Read our CMN interview with Dr. Morten Frödin and Professor Claus Storgaard Sørensen here.
CMN Articles - CRISPR-Select
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