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.
Other posts featuring Wolfgang Miesbach on Hemostasis Today.
-
Sep 23, 2026, 06:24Nicolas Gendron: Everolimus and Venous Thromboembolic Risk in Kidney Transplantation
-
Sep 23, 2026, 06:07Hoori Shahwar: Cerebral Venous Sinus Thrombosis Diagnosis and Management
-
Sep 23, 2026, 06:06Advancing Hemophilia Care and Patient Advocacy in Bangladesh – World Federation of Hemophilia
-
Sep 23, 2026, 05:54Bringing the Global Thrombosis and Haemostasis Community Together in 2027 – ISTH
-
Sep 23, 2026, 04:34David Akande: When Sickle Cell Crises Have No Clear Trigger
-
Sep 23, 2026, 04:13Jean Connors: Safety of Heparin Re-Exposure in Patients with Remote HIT
-
Sep 23, 2026, 04:09Beth Psaila: Applications Now Open for the Gordon Research Conference on Megakaryocytes and Platelets
-
Sep 23, 2026, 03:27Abbie Newton: How Better Data Can Reduce Platelet Wastage
-
Sep 23, 2026, 03:01Suvro Sankha Datta: Rapid Blood Group Genotyping Using Gold Oligo Nanonet Assembly