Blood reviews
Liver dysfunction in AAV-mediated Hemophilia B gene therapy: Mechanisms and management strategies.
Xuewen Song, Bingjie Ding, Hongkai Jia, Hu Zhou
Published: 202510.1016/j.blre.2025.101356
Abstract
Hemophilia B, an X-linked recessive bleeding disorder caused by mutations in the gene encoding coagulation factor IX (FIX). Gene therapy using adeno-associated viral (AAV) vectors targeting hepatocytes has emerged as a promising treatment, enabling s…
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