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…·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.