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Journal Articles

Real-world experience with apheresis for gene therapy in transfusion-dependent β-thalassemia: The largest single-center report

Vismay Deshani, Farhan Ali Anjum, Nandini Sadasivam, Ramya Hanasoge-Nataraj, Sabiha Kausar, Srividhya Senthil, Jaya Sudhir, Lucy Booth, Kelly Watts, Kiera Whitworth

Blood (2025) 146 (Supplement 1): 5964.

https://doi.org/10.1182/blood-2025-5964

Published: 2025

Journal Articles

Ex-vivo modification of autologous CD34+ HSPCs using a CD11B-directed lentiviral vector encoding APOEII-tagged human IDS leads to supraphysiological enzyme activity and early biochemical correction of neuronopathic mucopolysaccharidosis type II patients

Nathan Jeffreys, Claire Horgan, Kelly Watts, Kiera Whitworth, Heather Church, Stewart Rust, Claire Booth, Karen Buckland, Katharin Balbirnie-Cumming, Rebecca Holley, Brian Bigger, Simon Jones, R.F. Wynn

Blood (2025) 146 (Supplement 1): 384.

https://doi.org/10.1182/blood-2025-384

Published: 2025