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    University - Student Department: Stem Cell Operations Position: Graduate

All Publications

  • Myeloid cell replacement induces intercellular mitochondrial transfer and restores metabolism in a mouse model of mitochondrial disease. Nature communications Cho, H., Sayana, R., Koladiya, A., Colella, P., Cho, S., Jahng, J. W., Vasquez, J. J., Arozqueta Basurto, J., Wu, J. C., Gomez-Ospina, N. 2026; 17 (1)

    Abstract

    Friedreich's ataxia (FA) is a mitochondrial disease caused by frataxin deficiency that leads to progressive neurodegeneration and cardiomyopathy. Effective disease-modifying therapies remain limited. Here we show that myeloid cell replacement promotes neurological and cardiac recovery in FA mice through intercellular mitochondrial transfer. Donor-derived mitochondria are transferred from microglia and macrophages to central nervous system cells and cardiomyocytes, increasing oxidative phosphorylation and ATP synthesis gene expression and mitochondrial protein abundance. These molecular changes are accompanied by improved survival and growth in male and female mice and enhanced spontaneous locomotion, strength, coordination and cardiac and function in female mice. In cultured cells, mitochondrial transfer requires direct cell-cell contact and partially restores respiratory capacity in frataxin-deficient recipient cells, which exhibit enhanced mitochondrial uptake, suggesting disease-specific mechanisms that promote mitochondrial acquisition or retention. These findings identify mitochondrial transfer as a mechanism underlying the therapeutic effects of myeloid cell replacement and support hematopoietic transplantation for FA and other mitochondrial disorders.

    View details for DOI 10.1038/s41467-026-76775-y

    View details for PubMedID 42736305

    View details for PubMedCentralID PMC13574779

  • Hematopoietic stem cell transplantation for lysosomal disorders: Unraveling the mechanisms for CNS repopulation Arozqueta-Basurto, J., Poletto, E., Vera, L., Colella, P., Gomez-Ospina, N. ACADEMIC PRESS INC ELSEVIER SCIENCE. 2025: 5-6

    View details for DOI 10.1016/j.ymgme.2024.108641

    View details for Web of Science ID 001428859700034

  • CNS-wide repopulation by hematopoietic-derived microglia-like cells corrects progranulin deficiency in mice. Nature communications Colella, P., Sayana, R., Suarez-Nieto, M. V., Sarno, J., Nyame, K., Xiong, J., Pimentel Vera, L. N., Arozqueta Basurto, J., Corbo, M., Limaye, A., Davis, K. L., Abu-Remaileh, M., Gomez-Ospina, N. 2024; 15 (1): 5654

    Abstract

    Hematopoietic stem cell transplantation can deliver therapeutic proteins to the central nervous system (CNS) through transplant-derived microglia-like cells. However, current conditioning approaches result in low and slow engraftment of transplanted cells in the CNS. Here we optimized a brain conditioning regimen that leads to rapid, robust, and persistent microglia replacement without adverse effects on neurobehavior or hematopoiesis. This regimen combines busulfan myeloablation and six days of Colony-stimulating factor 1 receptor inhibitor PLX3397. Single-cell analyses revealed unappreciated heterogeneity of microglia-like cells with most cells expressing genes characteristic of homeostatic microglia, brain-border-associated macrophages, and unique markers. Cytokine analysis in the CNS showed transient inductions of myeloproliferative and chemoattractant cytokines that help repopulate the microglia niche. Bone marrow transplant of progranulin-deficient mice conditioned with busulfan and PLX3397 restored progranulin in the brain and eyes and normalized brain lipofuscin storage, proteostasis, and lipid metabolism. This study advances our understanding of CNS repopulation by hematopoietic-derived cells and demonstrates its therapeutic potential for treating progranulin-dependent neurodegeneration.

    View details for DOI 10.1038/s41467-024-49908-4

    View details for PubMedID 38969669

    View details for PubMedCentralID PMC11226701

  • Genome-edited hematopoietic stem cells as a curative approach for Gaucher disease type 1 Vera, L., Gastou, M., Arozqueta-Basurto, J., Colella, P., Feng, A., Poletto, E., Gomez-Ospina, N. ACADEMIC PRESS INC ELSEVIER SCIENCE. 2024

    View details for DOI 10.1016/j.ymgme.2023.107998

    View details for Web of Science ID 001192169300260

  • Transplantation without Myeloablation: Novel Conditioning Enables Robust Repopulation of Macrophage/Microglia Niches by Bone Marrow-Derived Cells Colella, P., Suarez-Nieto, V., Arozqueta-Basurto, J., Gomez-Ospina, N. CELL PRESS. 2022: 60-61

    View details for Web of Science ID 000794043700112