Exhibitor Tutorial

Innovative CRISPR/Cas9 gene knockin and SNP-detection tools for establishing human iPS-derived disease model lines for drug screening

Tuesday, February 6
2:00 PM - 2:45 PM
Location: Room 1A

The unique combination of precise, footprint-free editing using CRISPR/Cas9 and human induced pluripotent stem (hiPS) cells allows for a new level of sophistication in generating disease models which allow for rapid advancement in the development of new therapeutics. While CRISPR/Cas9-based gene editing is an effective technique to obtain knockout mutations with high efficiency, knocking in longer genes or sequences (>200 bp) via homology directed repair (HDR) is difficult to complete successfully. Therefore, more sophisticated screening tools are required for low efficiency knockins that can easily identify the edited clonal cell lines containing the engineered sequence.
One of the most powerful applications of genome editing is the introduction of base changes in specific genomic sites that mimic single-nucleotide polymorphisms (SNPs) related to human diseases or introducing stop codons to generate gene knockouts. Although, screening large number of clones to identify edited clonal cell lines containing the engineered base-of-interest is still a bottleneck, especially in the absence of a phenotypic readout.
To address this need, we developed a simple, high-throughput SNP-detection method that allows for rapid screening of clones from 96-well plates and detection of edited clonal cell lines independent of the engineered nucleotide substitution and the surrounding targeted genomic loci. As a proof-of-concept, we applied this method to successfully detect all the possible transitions in several human gene loci using genomic DNA as template or directly in cultured human fibroblasts. This screening method was then successfully used to screen hiPSCs clonal cell lines for SNPs related to tyrosinemia that were generated using CRISPR/Cas9.

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Liz Quinn

Associate Director for Stem Cell Marketing
Takara Bio USA, Inc.

Dr. Liz Quinn is the Associate Director for Stem Cell Marketing at Takara Bio USA following nearly 20 years of research and commercial experience in the life science and biotechnology industries. Dr. Quinn joined Clontech Laboratories, Inc. in 2001 and held varied responsibility within Operations, R&D and Marketing. In 2008, she joined DiscoverX Corporation and managed the GPCR drug discovery business. After leaving DiscoverX in 2014, she served as founder and CEO for a new life science and drug discovery-focused marketing consulting company based in Northern CA. Then in 2015, Liz joined Takara Bio USA where she has focused on launching a new stem cell product portfolio which includes human iPS and ES-derived lines and culture systems for gene editing. In addition to her current role, Liz is a board member of ELRIG, the European Laboratory Research and Innovation Group, and has served as their Scientific Programme Director since 2014.

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