The paper details the development of DNA-Diffusion, a generative artificial intelligence framework designed to engineer synthetic DNA regulatory elements. By utilizing a diffusion model trained on large-scale genomic data, researchers can now create compact sequences that dictate how genes are turned on or off in specific cell types. These lab-designed elements were shown to be functionally superior to natural sequences, offering higher activity and better precision across different tissues. The study validated these findings using large-scale screening and successfully edited a leukemia-protective gene within its natural environment. Ultimately, this technology provides a powerful new toolkit for precision medicine and the advancement of targeted gene therapies.

References:

• DaSilva L F, Senan S, Kribelbauer-Swietek J F, et al. Designing synthetic regulatory elements using the generative AI framework DNA-Diffusion[J]. Nature Genetics, 2025: 1-15.