Gastroenterology Unit, Department of Medicine, AOUI Policlinico G.B. Rossi and University of Verona, Piazzale L.A. Scuro 10, 37134, Verona, Italy.
San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Intern Emerg Med. 2019 Sep;14(6):911-921. doi: 10.1007/s11739-019-02126-z. Epub 2019 Jun 15.
Regenerative medicine represents the forefront of health sciences and holds promises for the treatment and, possibly, the cure of a number of challenging conditions. It relies on the use of stem cells, tissue engineering, and gene therapy alone or in different combinations. The goal is to deliver cells, tissues, or organs to repair, regenerate, or replace the damaged ones. Among stem-cell populations, both haematopoietic and mesenchymal stem cells have been employed in the treatment of refractory chronic inflammatory diseases with promising results. However, only mesenchymal stem cells seem advantageous as both systemic and local injections may be performed without the need for immune ablation. Recently, also induced pluripotent stem cells have been exploited for therapeutic purposes given their tremendous potential to be an unlimited source of any tissue-specific cells. Moreover, through the development of technologies that make organ fabrication possible using cells and supporting scaffolding materials, regenerative medicine promises to enable organ-on-demand, whereby patients will receive organs in a timely fashion without the risk of rejection. Finally, gene therapy is emerging as a successful strategy not only in monogenic diseases, but also in multifactorial conditions. Several of these approaches have recently received approval for commercialization, thus opening a new therapeutic era. This is why both General Practitioners and Internists should be aware of these great advancements.
再生医学代表了健康科学的前沿,有望治疗和(可能)治愈许多具有挑战性的疾病。它依赖于单独或不同组合使用干细胞、组织工程和基因疗法。其目标是输送细胞、组织或器官以修复、再生或替代受损的细胞、组织或器官。在干细胞群体中,造血干细胞和间充质干细胞都已被用于治疗难治性慢性炎症性疾病,并取得了有希望的结果。然而,只有间充质干细胞具有优势,因为可以进行全身和局部注射,而无需进行免疫消融。最近,诱导多能干细胞也被用于治疗目的,因为它们具有成为任何组织特异性细胞的无限来源的巨大潜力。此外,通过开发使用细胞和支持支架材料制造器官的技术,再生医学有望实现按需器官,使患者能够及时获得器官,而无需担心排斥反应。最后,基因治疗不仅在单基因疾病中,而且在多因素疾病中也成为一种成功的策略。最近,其中一些方法已获得商业化批准,从而开启了一个新的治疗时代。这就是为什么普通科医生和内科医生都应该了解这些重大进展。