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治愈糖尿病的先进疗法:“不可能完成的任务”现在有可能实现吗?

Advanced therapy to cure diabetes: mission impossible is now possible?

作者信息

Rohban Rokhsareh, Martins Christina P, Esni Farzad

机构信息

Department of Internal Medicine, Division of Hematology, Medical University of Graz, Graz, Austria.

Department of Surgery, Division of Pediatric General and Thoracic Surgery, Children's Hospital of Pittsburgh, University of Pittsburgh Medical Center, Pittsburgh, PA, United States.

出版信息

Front Cell Dev Biol. 2024 Nov 19;12:1484859. doi: 10.3389/fcell.2024.1484859. eCollection 2024.

Abstract

Cell and Gene therapy are referred to as advanced therapies that represent overlapping fields of regenerative medicine. They have similar therapeutic goals such as to modify cellular identity, improve cell function, or fight a disease. These two therapeutic avenues, however, possess major differences. While cell therapy involves introduction of new cells, gene therapy entails introduction or modification of genes. Furthermore, the aim of cell therapy is often to replace, or repair damaged tissue, whereas gene therapy is used typically as a preventive approach. Diabetes mellitus severely affects the quality of life of afflicted individuals and has various side effects including cardiovascular, ophthalmic disorders, and neuropathy while putting enormous economic pressure on both the healthcare system and the patient. In recent years, great effort has been made to develop cutting-edge therapeutic interventions for diabetes treatment, among which cell and gene therapies stand out. This review aims to highlight various cell- and gene-based therapeutic approaches leading to the generation of new insulin-producing cells as a topmost "panacea" for treating diabetes, while deliberately avoiding a detailed molecular description of these approaches. By doing so, we aim to target readers who are new to the field and wish to get a broad helicopter overview of the historical and current trends of cell- and gene-based approaches in β-cell regeneration.

摘要

细胞疗法和基因疗法被称为先进疗法,它们代表了再生医学的交叉领域。它们有相似的治疗目标,如改变细胞特性、改善细胞功能或对抗疾病。然而,这两种治疗途径存在重大差异。细胞疗法涉及引入新细胞,而基因疗法则需要引入或修饰基因。此外,细胞疗法的目的通常是替换或修复受损组织,而基因疗法通常用作预防手段。糖尿病严重影响患者的生活质量,并产生各种副作用,包括心血管疾病、眼科疾病和神经病变,同时给医疗系统和患者带来巨大的经济压力。近年来,人们为开发治疗糖尿病的前沿治疗干预措施付出了巨大努力,其中细胞疗法和基因疗法尤为突出。本综述旨在强调各种基于细胞和基因的治疗方法,这些方法可导致产生新的胰岛素生成细胞,作为治疗糖尿病的最主要“万灵药”,同时有意避免对这些方法进行详细的分子描述。通过这样做,我们旨在针对该领域的新手读者,希望他们能对基于细胞和基因的β细胞再生方法的历史和当前趋势有一个广泛的总体了解。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3711/11611888/6930e1db6cf3/fcell-12-1484859-g001.jpg

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