Synthetic XNA as bio-tool to eliminate genetic diseases in humans

Important results from the project

The project successfully met its initial goals, demonstrating the feasibility of using engineered polymerases with modified XNTPs. It also validated CSR as an effective selection method and yielded three promising polymerase variants. Beyond expectations, the project deepened understanding of how chemical modifications impact enzyme performance, laying the groundwork for future development of programmable XNA tools for treating genetic diseases.

Expected long term effects

The project is expected to have important long-term effects by enabling gene therapies using engineered XNA biomolecules. By overcoming key challenges in polymerase engineering for modified dNTPs, it lays the foundation for stable, programmable molecules that target genetic mutations precisely. Combined with improved delivery methods, this work could lead to safer, more effective, and potentially curative treatments for many genetic diseases, while advancing synthetic biology and diagnostics.

Approach and implementation

The project followed the original plan closely, with key activities—such as XNTP synthesis, polymerase testing, and CSR-based selection—executed as intended. The timeline was maintained, and the chosen approach proved suitable. While no new collaborations were formally established, the project sparked interest among our existing partners, particularly those active in non-viral gene therapy and in vivo gene editing. No external disruptions occurred, and the project delivered its intended results.