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Technical Note

Deep understanding of cell and gene therapy genome editing protocols enabled with single-cell sequencing

Abstract

Cell and gene therapies are yielding new treatments, which were inconceivable only a few decades ago, for genetic disorders including cancers and inherited diseases. Single-cell DNA sequencing on the Tapestri Platform provides the most sensitive and nuanced quantification of genetic edits made to single cells with a simple one-day high-throughput workflow. In this app note, we demonstrate the workflow and experimental designs for using single-cell DNA sequencing to analyze genetic edits, and discuss the results including quantification of on- and predicted off- target edits for multiple targets, zygosity, and translocations within in population of single cells.


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