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Gene Therapy Clears Early Hurdle in Deadly Childhood Liver Disease
Key Takeaways
- Researchers treated ARC syndrome in mice by delivering a healthy version of the gene linked to the disease.
- The final liver-targeted version avoided harms seen in earlier versions that triggered abnormal gene activation and cancerous growth.
- The team says more long-term safety work is needed before any human trial can begin.
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DT Editorial Team··via medicalxpress.com