Gene Therapy and Diabetes: A Narrative Review of Recent Advances and the Role of Multidisciplinary Healthcare Teams.

IF 2.8 3区 生物学 Q2 GENETICS & HEREDITY Genes Pub Date : 2025-01-20 DOI:10.3390/genes16010107
Nadia Khartabil, Ani Avoundjian
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Abstract

Introduction: Gene therapy has emerged as a promising frontier in the management of diabetes, offering innovative approaches to address both type 1 and type 2 diabetes. This narrative review examines the advancements in gene therapy applications, focusing on both animal and human studies, and includes a total of 11 studies in adherence to PRISMA guidelines. These studies utilize various viral vectors, such as adeno-associated virus (AAV) and lentivirus, to deliver genes that regulate insulin production and enhance angiogenesis. This review aims to synthesize recent advancements in gene therapy for both type 1 and type 2 diabetes and its complications, and to explore the evolving role of pharmacists in this emerging field. Methods: A comprehensive search was conducted to identify relevant studies on gene therapy for diabetes. Databases such as PubMed, the Cochrane Database of Systematic Reviews, the Cochrane Central Register of Controlled Trials, and Google Scholar were queried using keywords such as "Diabetes", "gene therapy", "Type 1 diabetes", and "Type 2 diabetes". Both animal and human studies were included to provide a broad perspective on the advancements in this field. Results: Animal model studies have shown promising results, including sustained insulin production, improved glucose homeostasis, and enhanced wound healing. Human studies, though fewer in number, have reported significant advancements. Patients with diabetic neuropathy treated with plasmid VEGF and recombinant adeno-associated virus (rAAV) showed improvements in neuropathic symptoms and glycemic control. Other studies involving intramuscular injections of VM202 and bicistronic VEGF165/HGF plasmid have reported pain reduction, improved healing of ischemic lesions, and increased angiogenesis. Conclusions: Despite these encouraging results, limitations such as small sample sizes, short follow-up periods, and the necessity for more extensive clinical trials persist. Diabetes is a metabolic syndrome that requires the collaboration of a multidisciplinary team to assist in several aspects of implementing successful gene therapy. Several healthcare providers and policy makers may play a crucial role in patient education, counseling, and the management of gene therapy treatments.

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基因治疗和糖尿病:最近的进展和多学科医疗团队的作用的叙述回顾。
基因治疗已成为糖尿病管理的一个有前途的前沿,为解决1型和2型糖尿病提供了创新的方法。这篇叙述性的综述考察了基因治疗应用的进展,重点是动物和人类的研究,并包括了11项遵循PRISMA指南的研究。这些研究利用各种病毒载体,如腺相关病毒(AAV)和慢病毒,来传递调节胰岛素产生和促进血管生成的基因。本文综述了1型和2型糖尿病及其并发症基因治疗的最新进展,并探讨了药师在这一新兴领域的作用。方法:综合检索糖尿病基因治疗的相关研究。使用“糖尿病”、“基因治疗”、“1型糖尿病”和“2型糖尿病”等关键词查询PubMed、Cochrane系统评价数据库、Cochrane中央对照试验登记册和谷歌Scholar等数据库。包括动物和人类的研究,以提供一个广阔的视角,在这一领域的进展。结果:动物模型研究显示了有希望的结果,包括持续的胰岛素产生,改善葡萄糖稳态,促进伤口愈合。人体研究虽然数量较少,但已经取得了重大进展。用质粒VEGF和重组腺相关病毒(rAAV)治疗的糖尿病神经病变患者显示出神经病变症状和血糖控制的改善。其他涉及肌肉注射VM202和双电VEGF165/HGF质粒的研究报道了疼痛减轻、缺血性病变愈合改善和血管生成增加。结论:尽管这些结果令人鼓舞,但样本量小、随访时间短以及需要更广泛的临床试验等局限性仍然存在。糖尿病是一种代谢综合征,需要多学科团队的合作,以协助在几个方面实施成功的基因治疗。一些医疗保健提供者和政策制定者可能在患者教育、咨询和基因治疗管理方面发挥关键作用。
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来源期刊
Genes
Genes GENETICS & HEREDITY-
CiteScore
5.20
自引率
5.70%
发文量
1975
审稿时长
22.94 days
期刊介绍: Genes (ISSN 2073-4425) is an international, peer-reviewed open access journal which provides an advanced forum for studies related to genes, genetics and genomics. It publishes reviews, research articles, communications and technical notes. There is no restriction on the length of the papers and we encourage scientists to publish their results in as much detail as possible.
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