Circular ssDNA boosts TALEN editing efficiency

“The implementation of these gene-editing techniques holds significant potential for the development of next-generation therapies”Beatriz Aranda Orgilles

In T-cells, CssDNA resulted in higher integration rates. In HSPCs, CssDNA outperformed LssDNA by improving transgene insertion frequency, cell viability, and insertion specificity.

Comparative transcriptomic analysis and long-term in vivo engraftment studies in highly immunodeficient NCG mice revealed that CssDNA delivery preserves HSPC fitness and supports more stable genome editing than AAV-based templates. These findings support the therapeutic relevance of CssDNA as a non-viral donor platform for safe and efficient gene insertion in clinically relevant cell types.

»The implementation of these gene-editing techniques holds significant potential for the development of next-generation therapies, aiming to provide alternative efficient, and safe therapeutic options for patients with cancer, autoimmune diseases, monogenic disorders, and various other conditions,« says Beatriz Aranda Orgilles, PhD, Associate Director at Cellectis and co-author of the study.

This work was conducted by researchers from Cellectis and Moligo Technologies and was recently presented at ASGCT 2025.