Disease background influences fate of transplanted stem cells

Gao, Teng; Sankaran, Vijay G.

doi:10.1038/d41586-024-03112-y

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23 October 2024

Disease background influences fate of transplanted stem cells

Gene-edited stem cells can be used in regenerative therapies to treat diverse genetic diseases. Tracking the output of these cells over time reveals a commitment to lineages that meet disease-specific needs.

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Teng Gao
1. Teng Gao is in the Division of Hematology and Oncology at Boston Children’s Hospital and Harvard Medical School, the Howard Hughes Medical Institute, and the Broad Institute of MIT and Harvard.
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Vijay G. Sankaran
1. Vijay G. Sankaran is in the Division of Hematology and Oncology at Boston Children’s Hospital and Harvard Medical School, the Howard Hughes Medical Institute, and the Broad Institute of MIT and Harvard.
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An emerging treatment option for genetic diseases that affect the blood and immune system is haematopoietic stem cell (HSC) gene therapy. In ‘autologous’ HSC gene therapy, a person’s own haematopoietic stem and progenitor cells (HSPCs) are collected and genetically modified to restore normal gene function, and then transplanted back into that person to re-establish the production of blood and immune cells (haematopoiesis) in the bone marrow. But what are the dynamics of haematopoietic regeneration by gene-corrected HSPCs, and how does this process differ between diseases? Writing in Nature, Calabria et al.¹ present a large-scale comparative study that tackles this question and uncovers the versatile ability of gene-corrected HSPCs to adapt to a person’s physiological needs, as determined by their disease background.

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Nature 636, 50-51 (2024)

doi: https://doi.org/10.1038/d41586-024-03112-y

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