TITLE:
A Review of the Research Progress on Stem Cell Therapy for Moyamoya Disease
AUTHORS:
Hanying Gu, Xiuxia Shi, Jiangtao Zhang
KEYWORDS:
Moyamoya Disease, Stem Cell Therapy, Mesenchymal Stem Cells (MSC), Induced Pluripotent Stem Cells (iPSC), Hematopoietic Stem Cells (HSC), Angiogenesis, Immunomodulation, Neuroprotection, Combination Therapy
JOURNAL NAME:
Journal of Biosciences and Medicines,
Vol.14 No.9,
September
2,
2026
ABSTRACT: Moyamoya disease (MMD) is a chronic cerebrovascular disease characterized by progressive stenosis and occlusion of major intracranial vessels and the formation of abnormal vascular networks at the base of the brain. Existing surgical revascularization procedures are ineffective in fundamentally halting the disease progression. Stem cell therapy, with its multiple biological functions, including promoting angiogenesis, immunomodulation, and neuroprotection, offers a novel treatment strategy for MMD. This article provides a comprehensive narrative review of the research progress on stem cell therapy for MMD, focusing on the mechanisms by which mesenchymal stem cells (MSC), induced pluripotent stem cells (iPSC), and hematopoietic stem cells (HSC) improve neurological function by paracrine secretion of vascular endothelial growth factor (VEGF) and other factors, promoting therapeutic angiogenesis, inhibiting the release of proinflammatory cytokines such as TNF-α and IL-6, and modulating connexin 43 (Cx43) expression. Preclinical studies have shown that stem cell transplantation can increase microvascular density and improve cerebral blood flow in MMD models. Preliminary clinical evidence suggests that autologous bone marrow stem cell mobilization combined with surgery can significantly reduce inflammatory cytokines and enhance cerebral blood flow reserve in patients. However, this field still faces challenges, including standardized stem cell preparation, long-term safety validation, and the lack of large-scale randomized controlled trials. At present, stem cell-based interventions for MMD remain investigational and should be considered adjunctive experimental approaches rather than established treatments. Future research can further explore aspects such as stem cell sources and quality control, survival efficiency, mechanism complexity, clinical research limitations, and safety to promote the clinical translation of stem cell therapy.