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poster

Precise Measurement of Viral Copy Number at Single-Cell Resolution for Cell and Gene Therapy Development


Matthew Cato

Cell and gene therapies (CGT) are transformative solutions for a host of inherited and acquired diseases for which existing interventions are ineffective. Many such therapies rely on the introduction of transgenes into host cells using viral or non-viral vectors. The accurate measurement of gene transfer is critical to the development of therapeutic agents and is a key attribute for assessing their safety and efficacy. Yet, conventional methods for measuring gene transfer lack the resolution and representation to truly reflect sample composition and either report a population average (bulk) or involve laborious and time-consuming clonal outgrowth which can take weeks. Mission Bio has developed an end-to-end solution from panel design to data analysis for single-cell targeted DNA sequencing. Here, using the Tapestri platform we demonstrate that single-cell DNA sequencing identifies transduced versus nontransduced cells with exceptional accuracy and precision, as well as measures the single-cell level vector copy number (VCN) for populations of thousands of cells with single nucleotide resolution while reducing sample processing time from weeks to days.


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poster
Single-cell Multi-omics Analysis Reveals Differential Lineage-specific Vector Copy Number Distribution in CAR-T Cell Products
Yilong Yang; Saurabh Parikh; Khushali Patel; Qawer Ayaz; Lindsey Murphy; Amanda Winters; Terry J. Fry; Mahir Mohiuddin; Hua-Jun He; John Elliott; Benjamin Schroeder; Shu Wang; Chieh-Yuan (Alex) Li
poster
High Throughput Single-cell Assessment of Genome Integrity and Toxicity Events Associated With Edited Cells
Chieh-Yuan (Alex) Li; Saurabh Parikh; Saurabh Gulati; Donjo Ban; Nechama Kalter; Michael Rosenburg; Qawer Ayaz; Joanne Nguyen; Benjamin Miltz; Yang Li1; Madhumita Shrikhande; Edward Szekeres; Ayal Hendel; Benjamin Schroeder; Shu Wang
poster
Single-cell Multiomic Clonal Tracking in Myeloma Identifies SMM Clones that Progress to MM and Low-Frequency MM Clones with Resistance Features Enabling More Precise Application of Targeted Therapies
Adam Sciambi
poster
A novel single-cell measurable residual disease (scMRD) assay for simultaneous DNA mutation and surface immunophenotype profiling
Holly Tillson
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