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.
Gene ReportsBiochemistry, 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.