Wolfgang Miesbach: Liver Organoids Advance the Future of AAV Gene Therapy Safety Testing
Wolfgang Miesbach, Professor of Medicine at Frankfurt University Hospital, shared a post on LinkedIn about recent article authored by Ritu M. Ramamurthy et al. published in Molecular Therapy Advances, adding:
“Liver organoids to stress-test AAV gene therapy
Impressive new data using a human liver tissue equivalent (hLTE) with hepatocytes, endothelial, stellate and Kupffer cells to evaluate liver-directed AAV vectors under human-relevant conditions.
In this platform, different AAV capsids and expression cassettes are compared for transduction efficiency, cell-type tropism, hepatotoxicity and inflammation, including readouts such as ALT/AST, albumin, A1AT and RNA-seq signatures.
Such organoid and liver-chip systems may become key tools to optimize dose and vector design and to better predict AAV safety before first-in-human trials in hemophilia and other monogenic liver diseases.
Great study by Ritu M. Ramamurthy, Graça Almeida-Porada and co-authors in Molecular Therapy Advances.”
Title: Defining the Safety and Efficacy of Liver-Directed AAV Gene Therapy Using a Human Liver Tissue Equivalent Platform
Authors: Ritu M. Ramamurthy, Wen Ting Zheng, Sarah E. Wachtman, Jonathan H. Diaz, Sunil K. George, Trang Simon, Yu Zhou, Meimei Wan, Baisong Lu, Stephen J. Walker, Colin E. Bishop, Christopher B. Doering, H. Trent Spencer, Anthony Atala, Christopher D. Porada, and Graça Almeida-Porada.
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