Leveraging Single-Cell DNA Sequencing For In-Depth Characterization Of Cell And Gene Therapies

By Jacqueline Marin, Benjamin Schroeder, Shu Wang, Daniel Mendoza, Adam Sciambi, Brittany Enzmann

CRISPR is a powerful tool for the development of novel cell and gene therapies. Despite its ability to precisely alter the genome, CRISPR can yield off-target edits and rare chromosomal alterations that can impact the safety and efficacy of therapeutic agents. For this reason, protocol optimization and in-depth characterization are critical to successfully bringing new products to market.

Conventional sequencing technologies that analyze CRISPR-edited cells often report bulk measurements, an approach that fails to measure the zygosity and co-occurrence of alterations within individual cells. Analysis of clonal populations provides this information but can add weeks to experimental timelines.

In this poster, we demonstrate that single-cell DNA sequencing on Mission Bio’s Tapestri Platform provides sensitive and nuanced quantification of genetic edits made to up to 10,000 individual cells with a simple workflow.