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August 24, 2023 by Sally Floyd 3 min read

Gene-Editing Based Gene Therapies: Advancements and Insights With Single-Cell Analysis

Gene-editing based gene therapies have emerged as revolutionary approaches for targeting and treating genetic diseases. These therapies utilize cutting-edge technologies like CRISPR to modify and correct disease-causing genetic mutations. To fully unlock their potential, it is crucial to understand the mechanism of action, efficiency, and safety of these therapies. This necessitates the precise measurement of delivery transduction efficiency and assessment of gene editing outcomes. The Tapestri Single-Cell Analysis platform offers an invaluable tool for making these measurements, enabling gene therapy developers to gain essential insights and drive advancements in the field.

Gene-Editing Based Gene Therapies: How They Work and Recent Breakthroughs

Gene-editing based gene therapies involve precisely targeting and modifying genetic material within a patient’s cells. The most widely used technique is CRISPR-Cas9, which utilizes guide RNA molecules to direct the Cas9 enzyme to specific gene sequences. Once at the target site, Cas9 induces a double-strand break in the DNA, triggering cellular repair mechanisms that can be harnessed to introduce desired genetic modifications.

Recent breakthroughs in gene editing have showcased remarkable advancements. For instance, gene therapy developers have employed CRISPR-based therapies to treat previously incurable genetic diseases like sickle cell anemia and beta-thalassemia. These breakthroughs have demonstrated the potential of gene editing to provide life-changing treatments for patients, ushering in a revolutionary era in medicine.

Why are Transduction Efficiency and Gene Editing Analysis Significant for Gene Therapies?

Gene transduction efficiency, the measure of how efficiently therapeutic genetic material reaches the target cells, is a critical parameter to evaluate in gene therapies. Understanding the transduction efficiency helps optimize delivery methods, determine appropriate dosing, and improve treatment efficacy. Accurate measurement of transduction efficiency allows researchers to assess the percentage of successfully modified cells and gauge the therapy’s overall effectiveness.

For gene editing-based therapies, it’s equally important to evaluate gene editing outcomes in target cells. This involves assessing the precision and accuracy of the intended genetic modifications and identifying any off-target effects. These measurements are more complicated to uncover, given the heterogeneity of editing outcomes that result in genome engineering techniques like CRISPR. These include zygosity differences, varying co-occurrence of multiplex edits, and off-target edits. Traditional bulk analyses may overlook the heterogeneity of edited cells and fail to capture these important details. In contrast, Tapestri offers a high-resolution view of gene editing outcomes, providing insights into the diversity of edited cell populations and enabling the identification of potential safety concerns.

The Role of Tapestri Single-Cell Analysis in Gene Therapy Development

Tapestri Single-Cell Analysis is a powerful platform that allows gene therapy developers to measure delivery transduction efficiency and assess gene editing outcomes at the single-cell level. Tapestri’s ability to analyze gene editing outcomes at the single-cell level provides crucial insights into the diversity of edited cell populations and the presence of off-target effects. This knowledge can enable gene therapy developers to optimize treatment strategies, refine editing techniques, and ensure the safety and precision of their therapies.

Gene-editing based gene therapies hold tremendous promise for treating a wide range of genetic diseases. Understanding transduction efficiency and gene editing outcomes is essential for unlocking their full potential. The Tapestri Single-Cell Analysis platform provides gene therapy developers with a powerful tool to make these measurements, offering high-resolution insights and driving advancements in the field. By harnessing the capabilities of Tapestri, gene therapy developers can optimize their treatments, improve patient outcomes, and revolutionize the way genetic diseases are treated.

Mission Bio recently announced a full solution for genome editing analysis, which will be launched later this year. The Tapestri Genome Editing Solution will provide a comprehensive workflow from target panel design, reagents, and streamlined bioinformatic analysis for simple reporting– making it easier than ever to glean insights about zygosity of edits, co-occurrence of multiplex edits, on- vs. off-target edits, in combination with surface protein expression.

If you are interested in learning more about how single-cell transduction efficiency measurements and genome editing analysis can enable your gene therapy development, please contact us!

You can also refer to these additional resources on the Genome Editing Solution and transduction efficiency assay:


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