Replication-defective retroviral vectors are a powerful tool for ex vivo gene transfer in human cells, offering efficient therapeutic potential for conditions such as hemoglobinopathies, skin adhesion disorders, immunodeficiencies, and neurometabolic diseases in cell and gene therapy applications. The genetically modified cells produced through these methods are classified as "medicinal products" and must adhere to strict safety and manufacturing regulations throughout production.
A critical aspect of designing safe lentiviral vectors involves evaluating key parameters, including the percentage of transduced cells, vector copy number per cell, and integration sites. Currently, these assessments rely on multiple separate assays, often performed in bulk, making the process resource-intensive, expensive, and prone to incomplete data.
To address these limitations, we have developed a high-throughput, droplet-based method that integrates all these analyses into a single assay. This approach streamlines the process, reducing costs while providing comprehensive, single-cell resolution data critical for safer and more efficient vector design and transduction.