
Gene therapy using adeno-associated virus (AAV) vectors shows promise for genetic and acquired disorders, including haemophilia, spinal muscular atrophy, and liver diseases. However, liver injury, ranging from mild enzyme elevation to acute failure, poses challenges. Causes include immune responses and direct toxicity, with variability across conditions. Haemophilia A patients are more susceptible to liver reactions than those with haemophilia B. Corticosteroids effectively manage liver reactions and protect therapeutic efficacy. This review highlights liver injury risks in gene therapy and outlines strategies for prevention and management.
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