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糖尿病的细胞疗法与再生策略——STEMSO会议论文集

Cellular Therapies and Regenerative Strategies for Diabetes - Proceeding of the STEMSO Conference.

作者信息

Ricordi C, Inverardi L, Pileggi A, Pastori R L, Dominguez-Bendala J

机构信息

Diabetes Research Institute and Cell Transplant Program, University of Miami Miller School of Medicine, Miami, FL, USA.

出版信息

CellR4 Repair Replace Regen Reprogram. 2014;2(1). Epub 2014 Feb 5.

Abstract

Diabetes is now considered a growing global epidemic with sizable negative effects on patients' quality of life and life expectancy, and escalating economic impact (41% growth in the past five year), now representing health care ex-penditure impact of 256 Billion/year in the US alone. Objectives of cellular therapies and regenerative medicine strategies for treatment of diabetes are to reverse the disease condition and prevent the development of the severe chronic complications that can affect most organ systems in a large proportion of patients over time. Cell based therapies include the combination of immunomodulatory approaches aimed at restoring self tolerance (i.e., in the case of autoimmune diabetes) and at inducing permanent acceptance of transplanted tissues (in the case of allogeneic donors), or immune protection (i.e., engineered microenvironment and/or encapsulation) so that the immune system can no longer destroy the new insulin producing cells introduced either by regenerating, reprogramming or replacement. Several approaches are currently under evaluation for restoration of beta cell mass. The prototype strategy for Replacement is pancreatic islet transplantation, which is now an approved procedure in several countries. Reprogramming from non insulin-producing cells or Regeneration strategies could represent an appealing alternative to overcome shortage of deceased donor organs for transplantation. The selection of the most appropriate source for insulin producing cells is still not defined and the selected alternatives between replacement, reprogramming and regeneration strategies will be further developed in pre-clinical model systems and pilot clinical trials, while carefully assessing safety, efficacy and cost-effectiveness, as well as the challenges imposed by scaling up the selected technologies to meet the demand of the millions of affected patients who could benefit from these strategies.

摘要

糖尿病如今被视为一种在全球范围内日益蔓延的流行病,对患者的生活质量和预期寿命产生了相当大的负面影响,且经济影响不断升级(在过去五年中增长了41%),仅在美国,目前每年的医疗保健支出影响就达2560亿美元。细胞疗法和再生医学治疗糖尿病的策略目标是逆转疾病状况,并预防严重慢性并发症的发生,这些并发症会随着时间的推移影响大部分患者的大多数器官系统。基于细胞的疗法包括免疫调节方法的组合,旨在恢复自身耐受性(例如,在自身免疫性糖尿病的情况下),诱导对移植组织的永久接受(在同种异体供体的情况下),或进行免疫保护(例如,构建微环境和/或封装),以便免疫系统不再破坏通过再生、重编程或替换引入的新的胰岛素产生细胞。目前正在评估几种恢复β细胞数量的方法。替代的原型策略是胰岛移植,目前在几个国家已成为一种获批的程序。从非胰岛素产生细胞进行重编程或再生策略可能是克服移植用已故供体器官短缺的一种有吸引力的替代方法。用于产生胰岛素细胞的最合适来源尚未确定,替代、重编程和再生策略之间的选定方案将在临床前模型系统和试点临床试验中进一步开发,同时仔细评估安全性、有效性和成本效益,以及扩大选定技术规模以满足数百万可能从这些策略中受益的受影响患者需求所带来的挑战。

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