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探索1型糖尿病的新型治疗方式:潜力与前景。

Exploring Novel Treatment Modalities for Type 1 Diabetes Mellitus: Potential and Prospects.

作者信息

Salama Rasha Aziz Attia, Patni Mohamed Anas Mohamed Faruk, Ba-Hutair Shadha Nasser Mohammed, Wadid Nihal Amir, Akikwala Mushirabanu Sharifmiyan

机构信息

Department of Community Medicine, College of Medicine, Ras Al Khaimah Medical and Health Science University, Ras Al Khaimah 11172, United Arab Emirates.

Kasr El Aini Faculty of Medicine, Cairo University, Giza 12525, Egypt.

出版信息

Healthcare (Basel). 2024 Jul 26;12(15):1485. doi: 10.3390/healthcare12151485.

Abstract

Despite the effectiveness of insulin injections in managing hyperglycemia in type 1 diabetes mellitus (T1DM), they fall short in addressing autoimmunity and regenerating damaged islets. This review aims to explore the potential and prospects of emerging treatment modalities for T1DM, including mesenchymal stem cells (MSCs), MSC-derived exosomes, gene therapy, islet allotransplantation, pancreatic islet cell transplantation, and teplizumab. We review emerging treatment modalities for T1DM, highlighting several promising strategies with varied mechanisms and outcomes. Mesenchymal stem cells demonstrate potential in modulating the immune response and preserving or restoring beta-cell function, although variability in sources and administration routes necessitates further standardization. Similarly, MSC-derived exosomes show promise in promoting beta-cell regeneration and immune regulation, supported by early-stage studies showing improved glucose homeostasis in animal models, albeit with limited clinical data. Gene therapy, utilizing techniques like CRISPR-Cas9, offers targeted correction of genetic defects and immune modulation; however, challenges in precise delivery and ensuring long-term safety persist. Islet allotransplantation and pancreatic islet cell transplantation have achieved some success in restoring insulin independence, yet challenges such as donor scarcity and immunosuppression-related complications remain significant. Teplizumab, an anti-CD3 monoclonal antibody, has demonstrated potential in delaying T1DM onset by modulating immune responses and preserving beta-cell function, with clinical trials indicating prolonged insulin production capability. Despite significant progress, standardization, long-term efficacy, and safety continue to pose challenges across these modalities. While these therapies demonstrate significant potential, challenges persist. Future research should prioritize optimizing these treatments and validating them through extensive clinical trials to enhance T1DM management and improve patient outcomes.

摘要

尽管胰岛素注射在控制1型糖尿病(T1DM)的高血糖方面有效,但在解决自身免疫和修复受损胰岛方面仍存在不足。本综述旨在探讨T1DM新兴治疗方式的潜力和前景,包括间充质干细胞(MSCs)、MSC衍生的外泌体、基因治疗、胰岛同种异体移植、胰岛细胞移植和替普珠单抗。我们回顾了T1DM的新兴治疗方式,重点介绍了几种具有不同机制和结果的有前景的策略。间充质干细胞在调节免疫反应以及维持或恢复β细胞功能方面显示出潜力,尽管来源和给药途径的变异性需要进一步标准化。同样,MSC衍生的外泌体在促进β细胞再生和免疫调节方面显示出前景,早期研究表明动物模型中的葡萄糖稳态得到改善,尽管临床数据有限。利用CRISPR-Cas9等技术的基因治疗可实现基因缺陷的靶向纠正和免疫调节;然而,在精确递送和确保长期安全性方面的挑战仍然存在。胰岛同种异体移植和胰岛细胞移植在恢复胰岛素自主性方面取得了一些成功,但供体稀缺和免疫抑制相关并发症等挑战仍然很大。替普珠单抗是一种抗CD3单克隆抗体,通过调节免疫反应和维持β细胞功能,在延迟T1DM发病方面显示出潜力,临床试验表明其胰岛素产生能力延长。尽管取得了重大进展,但在这些治疗方式中,标准化、长期疗效和安全性仍然是挑战。虽然这些疗法显示出巨大潜力,但挑战依然存在。未来的研究应优先优化这些治疗方法,并通过广泛的临床试验对其进行验证,以加强T1DM的管理并改善患者预后。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/20d5/11311856/3fe158f69491/healthcare-12-01485-g001.jpg

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