Gentili Chiara, Torre Michele, Cancedda Ranieri
Department of Experimental Medicine (DIMES), University of Genoa, Laboratory of Regenerative Medicine, Genova, Italy.
Department of Pediatric Surgery, Istituto Giannina Gaslini, Genova, Italy.
Eur J Pediatr Surg. 2014 Jun;24(3):263-9. doi: 10.1055/s-0034-1382777. Epub 2014 Jun 22.
The therapeutic use of stem cells is a very promising strategy in the area of regenerative medicine. The stem cell regenerative paradigm has been mostly based on the assumption that progenitor cells play a critical role in tissue repair by their plasticity and differentiation potential. However, recent works suggest that the mechanism underlying the benefits of stem cell transplantation might relate to a paracrine modulatory effect rather than the replacement of affected cells at the site of injury. Preclinical and clinical skeletal studies, conducted in animal and adult series, support the use of mesenchymal stem cells (MSCs) for bone healing in critical clinical situations. These results have led to an increasing number of papers reporting the use of MSCs in adult clinical trials, whereas only few papers reported the use of these cells in pediatric skeletal disorders, probably because of unknown long-term results and long-life consequences of cellular therapy. The exponential growth of knowledge in adult MSCs could be translated and applied to pediatric disorders. Pediatric osteoarticular diseases have an enormous potential to be treated by MSCs, as severe congenital bone or local cartilage defects, not responding to conventional surgery treatment, might be successfully treated by cellular therapy. Translating basic stem cell research into routine therapies is a complex multistep process which entails the managing of the expected therapeutic benefits with the potential risks in correlation within the existing regulations. Here, we reported the state of art on the use of MSC in skeletal pediatric disorders.
干细胞的治疗应用是再生医学领域一项非常有前景的策略。干细胞再生模式主要基于这样一种假设,即祖细胞凭借其可塑性和分化潜能在组织修复中发挥关键作用。然而,最近的研究表明,干细胞移植益处的潜在机制可能与旁分泌调节作用有关,而非损伤部位受影响细胞的替代。在动物和成人系列中进行的临床前和临床骨骼研究支持在关键临床情况下使用间充质干细胞(MSC)促进骨愈合。这些结果导致越来越多的论文报道在成人临床试验中使用MSC,而只有少数论文报道在儿童骨骼疾病中使用这些细胞,这可能是由于细胞治疗的长期结果和长期影响尚不清楚。成人MSC知识的指数级增长可以转化并应用于儿童疾病。儿童骨关节疾病有很大的潜力通过MSC进行治疗,因为严重的先天性骨或局部软骨缺陷对传统手术治疗无反应,可能通过细胞治疗成功治愈。将基础干细胞研究转化为常规疗法是一个复杂的多步骤过程,需要在现有法规范围内管理预期的治疗益处与潜在风险。在此,我们报告了MSC在儿童骨骼疾病中应用的现状。