Cellectis, a biotechnology company, recently showcased groundbreaking research at the ASGCT Annual Meeting, unveiling advancements in non-viral gene editing and base editing technologies. The company’s innovative approach combines TALEN®-mediated gene editing with non-viral delivery of transgenes to propel cellular and gene therapies forward. This research, presented in two posters, highlights the potential of these technologies to revolutionize disease treatment by modifying disease-associated genes, offering new therapeutic avenues for various conditions.
By leveraging non-viral methods for gene editing, Cellectis demonstrated that TALEN®-mediated gene editing with non-viral templates could efficiently insert genes in T-cells and hematopoietic stem and progenitor cells (HSPCs), promoting viability and insertion specificity. This approach offers advantages over traditional viral vectors, showing better maintenance of cellular fitness and more stable gene editing using circular ssDNA templates. These findings hold promise for developing next-generation therapies for cancer, autoimmune diseases, and other disorders.
Another aspect of Cellectis’ research focused on high fidelity C-to-T editing using TALE base editors (TALEB). These editors, which convert cytosine to thymine, were studied for their efficiency and specificity in gene editing. The research explored factors influencing TALEB activity and assessed the editing outcomes, emphasizing the precision and efficiency of these editors in primary T cells. The results enhance the control and applicability of TALEB for future therapeutic uses.
The development of these gene-editing techniques marks a significant step forward in the field of biotechnology, offering new tools for precise and efficient gene editing. Beatriz Aranda Orgilles, an expert at Cellectis, emphasized the potential of these advancements in providing safe and effective therapeutic options for a wide range of medical conditions. Louisa Mayer, another Cellectis scientist, highlighted the efficiency and specificity of TALEB technology, underscoring its value in supporting novel gene editing and therapy applications.
Overall, Cellectis’ research at the ASGCT Annual Meeting showcases the company’s commitment to advancing gene-editing technologies for the benefit of patients. By harnessing the power of non-viral gene editing and base editing innovations, Cellectis is at the forefront of developing cutting-edge therapies that could transform the landscape of healthcare. As these technologies continue to evolve, they hold the promise of offering more targeted and personalized treatment options for patients worldwide.
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