The future of diabetes mellitus management: Therapeutic insights from gene and stem cell research

IF 0.9 Q4 GENETICS & HEREDITY
Snigdha Suman Das , E. Vignesh Balaji , K. Sreedhara Ranganath Pai
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引用次数: 0

Abstract

Aim

This review will explore the therapeutic potential of gene and stem cell therapy in diabetes mellitus, focusing on the latest developments in CRISPR/Cas9 gene editing, stem cell generated pancreatic beta cells and the hybrid therapeutic approaches in between.

Methods

A thorough review of the literature was performed, with the focus being on the mechanisms of gene therapy and stem cell differentiation and their integration with bioengineering technologies. Furthermore, therapeutic targets were identified and clinical trial outcomes were reviewed to determine the translational feasibility of these approaches.

Results

Gene therapy has shown potential in regenerating pancreatic beta cells and modulating immune responses through CRISPR/Cas9 based editing and insulin gene transfer. Stem cell based therapies have successfully differentiated into glucose responsive beta cells, and have reduced the need for insulin and enhanced glycemic control. The hybrid gene and stem cell based approaches have shown better durability and immune compatibility in the results and the 3D bioprinting is gradually developing implantable pancreatic tissue constructs. However, there are still some problems to be addressed, including immune rejection, ethical issues and the lack of scalability.

Conclusion

Gene and stem cell based interventions are potentially transformative in the management of diabetes, acting on the underlying pathophysiology rather than on symptoms only. These therapeutic modalities when integrated with bioengineering innovations, hold promise for developing curative approaches. However, safety, efficacy and ethical considerations of these critical challenges must be addressed in order to enable widespread clinical implementation of these advancements.
糖尿病管理的未来:来自基因和干细胞研究的治疗见解
目的探讨基因和干细胞治疗糖尿病的治疗潜力,重点介绍CRISPR/Cas9基因编辑、干细胞生成胰腺β细胞及其混合治疗方法的最新进展。方法对相关文献进行综述,重点介绍基因治疗和干细胞分化的机制及其与生物工程技术的结合。此外,还确定了治疗靶点,并对临床试验结果进行了回顾,以确定这些方法的转化可行性。结果基因治疗已经显示出通过基于CRISPR/Cas9的编辑和胰岛素基因转移来再生胰腺细胞和调节免疫反应的潜力。基于干细胞的治疗已经成功地分化为葡萄糖反应性β细胞,并减少了对胰岛素的需求,增强了血糖控制。结果表明,基于基因和干细胞的杂交方法显示出更好的耐久性和免疫相容性,生物3D打印正在逐步发展可植入的胰腺组织结构。然而,仍有一些问题需要解决,包括免疫排斥、伦理问题和缺乏可扩展性。结论基于基因和干细胞的干预在糖尿病的治疗中具有潜在的变革性,它们作用于潜在的病理生理而不仅仅是症状。这些治疗方式与生物工程创新相结合,有望开发出治疗方法。然而,这些关键挑战的安全性、有效性和伦理考虑必须得到解决,以便使这些进步能够广泛的临床实施。
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来源期刊
Gene Reports
Gene Reports Biochemistry, Genetics and Molecular Biology-Genetics
CiteScore
3.30
自引率
7.70%
发文量
246
审稿时长
49 days
期刊介绍: Gene Reports publishes papers that focus on the regulation, expression, function and evolution of genes in all biological contexts, including all prokaryotic and eukaryotic organisms, as well as viruses. Gene Reports strives to be a very diverse journal and topics in all fields will be considered for publication. Although not limited to the following, some general topics include: DNA Organization, Replication & Evolution -Focus on genomic DNA (chromosomal organization, comparative genomics, DNA replication, DNA repair, mobile DNA, mitochondrial DNA, chloroplast DNA). Expression & Function - Focus on functional RNAs (microRNAs, tRNAs, rRNAs, mRNA splicing, alternative polyadenylation) Regulation - Focus on processes that mediate gene-read out (epigenetics, chromatin, histone code, transcription, translation, protein degradation). Cell Signaling - Focus on mechanisms that control information flow into the nucleus to control gene expression (kinase and phosphatase pathways controlled by extra-cellular ligands, Wnt, Notch, TGFbeta/BMPs, FGFs, IGFs etc.) Profiling of gene expression and genetic variation - Focus on high throughput approaches (e.g., DeepSeq, ChIP-Seq, Affymetrix microarrays, proteomics) that define gene regulatory circuitry, molecular pathways and protein/protein networks. Genetics - Focus on development in model organisms (e.g., mouse, frog, fruit fly, worm), human genetic variation, population genetics, as well as agricultural and veterinary genetics. Molecular Pathology & Regenerative Medicine - Focus on the deregulation of molecular processes in human diseases and mechanisms supporting regeneration of tissues through pluripotent or multipotent stem cells.
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