As genome-editing systems move rapidly from the bench to advancing cell therapies, cellular-level understanding of complex systems is the key to informed success. En route to clinical applications, edited cellular systems need to be thoroughly characterized to fully understand the exact nature of induced mutations. Both on-target and off-target effects are equally important to detect and accurately quantify.
At Mission Bio we focus on the measurements and standards needed to increase confidence and lower the risk of utilizing genome-editing technologies in research and commercial products. Therefore, from development through production, careful analysis of edited cellular systems is a priority for those looking to bring exciting ex vivo and in vivo therapeutics to the clinic. The Tapestri Platform is a solution for high-throughput single-cell DNA analysis, delivering high-quality variant calling of single nucleotide variants (SNVs) and indels. Leveraging droplet microfluidics and a novel two-step workflow, high-multiplex PCR, cell barcoding, and targeted amplification is performed on tens of thousands of individual cells simultaneously.
Here we demonstrate the value of the Tapestri Platform for high resolution quality control for genome-editing systems. A Tapestri Single-Cell DNA Custom Panel was designed to measure the efficiency of a multiplex genome-editing system targeting three different loci in primary human T cells. Amplicons for known off-target sites and negative control sites were also included in the custom panel design.