1285-Lentiviral Gene Therapy for ARC Syndrome Treatment
This article investigates lentiviral gene therapy as a potential treatment for ARC syndrome, a fatal genetic disorder causing liver disease and multisystem failure. Researchers developed a liver-specific vector designed to restore the missing VPS33B protein, comparing its safety and efficacy against a ubiquitous vector in mouse models. The study discovered that while both vectors could correct cellular defects, the ubiquitous version carried a significant risk of liver tumors due to insertional oncogenesis. In contrast, the targeted liver-specific therapy proved safe and effective…
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