fig1

Gene therapy for inborn errors of immunity: past progress, current status and future directions

Figure 1. Autologous Haematopoietic Stem Cell Gene Therapies. Autologous HSC-GT involves harvesting the stem cells directly from the patient, which are then modified by gene addition or gene editing. Modified patient stem cells are then re-infused back into the patient, who in the meantime has undergone a conditioning regimen to deplete their endogenous immune cells. Following engraftment of the modified stem cells into the bone marrow of the patient, the newly introduced cells can repopulate the immune system with non-diseased cells. Created in BioRender. Torrance R (2025) https://BioRender.com/dmazkdh. HSC-GT: Haematopoietic stem cell gene therapies.

Rare Disease and Orphan Drugs Journal
ISSN 2771-2893 (Online)
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