Lang Erica, Semon Julie A
Department of Biological Sciences, Missouri University of Science and Technology, 400 W 11th St., Rolla, MO, USA.
Cell Regen. 2023 Feb 2;12(1):7. doi: 10.1186/s13619-022-00146-3.
Osteogenesis imperfecta (OI) is a disease caused by mutations in different genes resulting in mild, severe, or lethal forms. With no cure, researchers have investigated the use of cell therapy to correct the underlying molecular defects of OI. Mesenchymal stem cells (MSCs) are of particular interest because of their differentiation capacity, immunomodulatory effects, and their ability to migrate to sites of damage. MSCs can be isolated from different sources, expanded in culture, and have been shown to be safe in numerous clinical applications. This review summarizes the preclinical and clinical studies of MSCs in the treatment of OI. Altogether, the culmination of these studies show that MSCs from different sources: 1) are safe to use in the clinic, 2) migrate to fracture sites and growth sites in bone, 3) engraft in low levels, 4) improve clinical outcome but have a transient effect, 5) have a therapeutic effect most likely due to paracrine mechanisms, and 6) have a reduced therapeutic potential when isolated from patients with OI.
成骨不全症（OI）是一种由不同基因突变引起的疾病，可导致轻度、重度或致死性形式。由于无法治愈，研究人员已研究使用细胞疗法来纠正OI的潜在分子缺陷。间充质干细胞（MSCs）因其分化能力、免疫调节作用以及迁移至损伤部位的能力而备受关注。MSCs可以从不同来源分离，在培养中扩增，并且在众多临床应用中已被证明是安全的。这篇综述总结了MSCs治疗OI的临床前和临床研究。总的来说，这些研究的结果表明，来自不同来源的MSCs：1）在临床上使用是安全的，2）迁移至骨的骨折部位和生长部位，3）低水平植入，4）改善临床结果但具有短暂效应，5）最有可能通过旁分泌机制产生治疗效果，6）从OI患者中分离时治疗潜力降低。
