Modeling Adeno-Associated Viral Vector 6-mediated In Vivo Gene Delivery to Expanded Non-Mobilized Haemopoietic Stem Cells from Transfusion dependent Thalassemia Patients in a Humanized Mouse
Janani Ramesh, Karthikeyan Kandasamy, Nur Nazneen Binte Yusof, I. Sujuandy, Choong Tat Keng, Liwei Chen, Bing Shao Chia, Min Liu, Zhisheng Her, Marek Kukumberg, Kian Chuan Sia, Siti Humairah Mohd Rodhi, Zhen Ying Fu, Abdul Jalil Rufaihah, Alfredo Franco‐Obregón, , Citra Nurfarah Zaini Mattar
This study demonstrates the feasibility of using dual-ssAAV6 vectors for in vivo gene delivery to HSC from thalassemia patients in a humanized mouse model, showing efficient transduction and long-term persistence.
bioRxiv · Jun 17, 2025 · PreprintMolecular Biology