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Proper coupling between these two processes is essential for maintaining bone homeostasis, whereas its disruption leads to skeletal pathology. Excessive osteoclast activity underlies disorders such as osteoporosis and Paget\u2019s disease, while osteoclast deficiency, either quantitative or functional, results in osteopetrosis. Osteopetrosis comprises a group of genetically heterogeneous and clinically variable rare metabolic bone diseases characterized by increased bone mass. Clinically, osteopetrosis is commonly classified into severe autosomal recessive osteopetrosis (ARO), also known as infantile malignant osteopetrosis, intermediate ARO, milder autosomal dominant osteopetrosis, and X-linked recessive osteopetrosis. Effective treatment options for both severe and milder forms of osteopetrosis remain an unresolved clinical challenge. This review sheds light on the potential of cell-based and gene-modified cell therapies as emerging strategies for the treatment of osteopetrosis. 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