ReportsNeurodegenerative Disease

Transplantation of wild-type mouse hematopoietic stem and progenitor cells ameliorates deficits in a mouse model of Friedreich’s ataxia

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Science Translational Medicine  25 Oct 2017:
Vol. 9, Issue 413, eaaj2347
DOI: 10.1126/scitranslmed.aaj2347

Cell therapy for Friedreich’s ataxia

Friedreich’s ataxia (FRDA) is a lethal hereditary disease characterized by ataxia, neurodegeneration, muscle weakness, and cardiomyopathy and for which there is no treatment. Using a mouse model of FRDA, Rocca et al. show that wild-type hematopoietic stem and progenitor cell (HSPC) transplantation could lead to the rescue of the disease phenotype, including locomotor defects and muscle weakness. In addition, mitochondrial protein dysfunction was restored in the brain, skeletal muscle, and heart of the FRDA mice, potentially through transfer of mitochondrial proteins from HSPC-derived phagocytic cells to FRDA neurons and muscle myocytes.