Targeted Microglia Replacement Shows Promise Against Fatal Neurodegenerative Conditions

Targeted microglia replacement shows promise against fatal neurodegenerative conditions

Tay-Sachs and Sandhoff diseases, genetic disorders affecting the brain, have no effective treatment and are typically fatal within the first years of life.

Replacing brain immune cells in mice slows neurodegeneration: Technique may help with diseases like Alzheimer's

Tay-Sachs and Sandhoff diseases, genetic disorders affecting the brain, have no effective treatment and are typically fatal within the first years of life.

Therapeutic genetic restoration through allogeneic brain microglia replacement

Migration of transplanted allogeneic myeloid cells into the brain following systemic hematopoietic stem and progenitor cells transplantation (HCT) holds great promise as a therapeutic modality to correct genetic deficiencies in the brain such as lysosomal storage diseases.1–3 However, the toxic myeloablation required for allogeneic HCT can cause serious, life-threatening side effects limiting its applicability. Moreover, transplanted allogeneic …

Microglia Replacement Strategy Mitigates Rare Neurodegenerative Disease in Mice

Genetic neurodegenerative disorders like Tay-Sachs and Sandhoff disease currently have no effective treatments. A possible strategy is replacing affected cells with healthy ones in order to slow the neurodegenerative process. But this approach is not without challenges, including poor engraftment in the brain and a graft-versus-host response.  Now scientists from Stanford University School of Medicine, University Hospital Hamburg-Eppendorf, and …