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Michelle Ma

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Open access Jul 2026

Base Editing Hematopoietic Stem/Progenitor Cell Gene Therapy for Treatment of X-Linked Severe Combined Immunodeficiency

It is concluded from preliminary data that gene therapy using BE HSPCs appear very promising for achieving an earlier and more robust multi-lineage immune reconstitution in adult patients using BE HSPCs.

S. D. de Ravin, Michelle Ma, Yuzhi Yin et al. · 0 citations