Article
Inherent hepatocytic heterogeneity determines expression and retention of edited F9 alleles post-AAV/CRISPR infusion.
Proceedings of the National Academy of Sciences of the United States of America - 19 Oct 2021
Wang Qiang, Zhang Lin, Zhang Guo-Wei, Mao Jian-Hua, Xi Xiao-Dong, Jiang Lu, Lv Gang, Lu Jing, Shen Yan, Chen Zhu, Zhu Jiang, Chen Sai-Juan
Abstract excerpt
Infusing CRISPR/donor-loaded adeno-associated viral vectors (AAV/CRISPR) could enable in vivo hepatic gene editing to remedy hemophilia B (HB) with inherited deficiency of clotting factor IX (FIX). Yet, current regimens focus on correcting HB with simple mutations in the coding region of the F9, overlooking those carrying complicated mutations involving the regulatory region. Moreover, a possible adverse effect...
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