Design and validation of a GMP stem cell manufacturing protocol for MPSII hematopoietic stem cell gene therapy

Stuart Ellison, K. Buckland, Y. Learmonth, Victoria Day, Spandan Kalra, Lauren Howe, F. Román-Rodríguez, Jose Bonafont, Laura Booth, Rebecca Holley, Jon Smythe, Simon Jones, Adrian Thrasher, Claire Booth, Brian Bigger
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设计并验证用于 MPSII 造血干细胞基因疗法的 GMP 干细胞制造方案
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