PALERMO – Italian hematological research marks a historic milestone. The 2025 D'Agostino Prize for Innovative Research in Hematological Rare Diseases has been awarded to Professor Dario Balestra, professor of Molecular Biology at the University of Ferrara, and his research team for a groundbreaking study that opens new perspectives in the treatment of hemophilia A.
The work, entitled Base and Prime Editing: Precise DNA Tools Opening New Paths for Treating Hemophilia A, demonstrated for the first time the effectiveness of "base editing" and "prime editing" techniques in precisely correcting the genetic mutations responsible for the disease. These so-called "molecular scissors" repaired up to 20% of the defects in cellular models, restoring production of the missing clotting factor VIII and overcoming the limitations of current therapies, which require lifelong infusions.
The award, presented by an international committee of experts in rare hematological diseases, recognized the scientific merit and innovation of a strategy that could radically transform the therapeutic approach to hemophilia and other genetic blood disorders. "Receiving this recognition is a fundamental encouragement to continue toward the goal of curative therapy," stated Professor Balestra.
The research will now move toward preclinical testing on endothelial cells derived from patients and animal models, crucial steps toward potential clinical application. The Cutino Foundation, sponsor of the award, thus confirms its mission to support cutting-edge science and offer new, concrete hope to patients with rare hematological diseases.
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Comments (1)
This is a great step forward for hematology research, but I hope that these methods, which have proven effective, can also be applied to other genetic diseases and not just hemophilia A. Science moves forward.