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Precise Measurement of Transduction Efficiency at Single-Cell Resolution for Cell and Gene Therapy Development


Khushali Patel

Cell and gene therapies are transformative solutions for diseases where 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 such therapies and is critical for assessing their safety and efficacy. Yet, conventional methods for measuring gene transfer either report a population average (bulk) or involve laborious and time-consuming clonal outgrowth which can take up to 14 days. Mission Bio has developed an end-to-end solution from panel design to data analysis for single-cell targeted DNA sequencing. Using the Tapestri platform we demonstrate that single-cell DNA sequencing identifies transduced versus non-transduced cells with exceptional accuracy and precision for populations of up to 10,000 cells 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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