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DiffronC is an oligonucleotide-based gene therapy developed by Regen BioPharma that utilizes proprietary small interfering RNA (siRNA) technology to induce differentiation of cancer stem cells. Its mechanism involves silencing nuclear receptor targets—specifically those that maintain stem cells in an undifferentiated state—thereby promoting the maturation of blood-forming stem and progenitor cells into functional blood cells. This approach is designed as a differentiation therapy for hematological malignancies such as leukemia and myelodysplastic syndromes where normal cellular maturation is blocked. Preclinical data have demonstrated its ability to drive differentiation in vitro and provide proof-of-concept for its use in treating disorders characterized by impaired blood cell development[8][10][6].
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