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In an ASS1 low sarcoma in vivo model reported, ADI-PEG20 showed significant reduction in tumor growth compared to control, but a slow increase in tumor size was still observed over time and the tumor lysate showed significantly enhanced ASS1 expression [133]

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While thalassemia can be managed by lifelong blood transfusions and iron chelation therapy, or potentially cured by bone marrow transplantation or cord blood transfusions, none of these interventions are without side effects including neurotoxicity, cancer, and even death (Noe 2010