Transplanted allogeneic myeloid cell migration into the brain can correct genetic deficiencies but is limited by toxic myeloablation and immune rejection. A new approach using Sca1- committed progenitor cells allows for microglia replacement without preconditioning, circumventing peripheral engraftment and avoiding complications associated with graft-vs-host disease. This technique successfully rescues a murine model of Sandhoff disease. Furthermore, human induced pluripotent stem cell-derived myeloid progenitor cells show similar engraftment capabilities, suggesting significant potential for allogeneic microglial therapies in treating brain disorders.
Migration of transplanted allogeneic myeloid cells into the brain following systemic hematopoietic stem and progenitor cell transplantation holds promise for treating genetic deficiencies.
Myeloablative preconditioning limits the applicability of allogeneic HCT due to serious side effects and the vulnerability of transplanted cells to rejection.
Sca1- committed progenitor cells effectively replace microglia in the brain without the need for hematopoietic stem cells, leading to significant advancements in therapy.
The allogeneic microglia replacement method demonstrated therapeutic success by rescuing murine models of Sandhoff disease, indicating a breakthrough for treating lysosomal storage diseases.
#microglia-replacement #hematopoietic-stem-cells #lysosomal-storage-diseases #gene-therapy #allogeneic-transplantation
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