AAV8 Gene Therapy’s Unseen Risk: Triggering Cholestatic Liver Disease
A new study in Science Translational Medicine reveals a significant safety concern for gene therapy in a mouse model of X-linked myotubular myopathy. Researchers found that adeno-associated virus serotype 8 (AAV8)-based gene therapy, when combined with specific dietary insults, can precipitate cholestatic liver disease. This finding highlights a critical interaction between therapeutic intervention and environmental or metabolic stressors, suggesting that patient liver health and diet may be crucial variables in determining the safety profile of systemic AAV treatments.
Why it might matter to you: For gastroenterologists and hepatologists, this research underscores the importance of pre-therapeutic liver assessment and patient counseling in the era of advanced genetic treatments. It directly connects to the management of cholestasis, bile acid metabolism, and drug-induced liver injury, expanding the differential for liver dysfunction in patients undergoing novel therapies. This insight could inform clinical trial design and post-marketing surveillance for AAV-based treatments targeting a range of conditions.
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