Treating Lysosomal Storage Diseases Using iPSC-derived Microglia

Time: 3:30 pm
day: Day Two


Virtual Presentation

  • Inherited, lysosomal storage diseases (LSDs) are characterized by the pathologic build-up of toxic material in the body’s cells because of a genetic defect resulting in enzyme deficiencies.
  • Microglia, the resident myeloid cells of the brain, are dispersed throughout the brain and microglia progenitors can readily be derived from human induced pluripotent stem cells (hiPSCs); BlueRock identified them as a potential cell therapy for LSDs
  • We show that wildtype donor microglia progenitors can cross-correct the enzymatic deficiency in several LSDs in vitro and in vivo by transferring the missing enzyme to diseased cells