Padina Pahlavan,1,*Abbas Abbasi Rouholahi,2
1. Bachelor's student, Microbiology group, Faculty of Biology, University of Tehran, Tehran, Iran. 2. Microbiology and Biotechnology Laboratory, Department of Microbiology - Faculty of Biology - Science Campus - University of Tehran, Tehran, Iran.
Introduction: Type 1 Diabetes (T1D) constitutes an autoimmune disorder that precipitates the obliteration of insulin-secreting β-cells within the pancreatic tissue. Existing therapeutic modalities, predominantly comprising insulin replacement, fail to target the fundamental etiology and may lead to various complications. CRISPR/Cas9 represents an advanced genome-editing apparatus that employs a guide RNA (gRNA) to accurately direct the Cas9 nuclease towards designated DNA sequences, engendering double-strand breaks. These breaks are amenable to cellular repair mechanisms, thus permitting gene knockout or insertion. The advent of CRISPR technology presents a novel and promising paradigm for the management of T1D, facilitating precise genetic modifications aimed at promoting both β-cell regeneration and modulation of the immune response.
Methods:
To gain a comprehensive understanding of the role of CRISPR in Type1 Diabetes, a thorough literature search was conducted across PubMed, Google Scholar, and NCBI databases. This search identified 19 relevant articles that were carefully reviewed and analyzed to provide a deeper insight into this subject.
Methods: To gain a comprehensive understanding of the role of CRISPR in Type1 Diabetes, a thorough literature search was conducted across PubMed, Google Scholar, and NCBI databases. This search identified 19 relevant articles that were carefully reviewed and analyzed to provide a deeper insight into this subject.
Results: β-Cell Regeneration: Investigators are probing the application of CRISPR to modify stem cells with the objective of differentiating them into insulin-secreting β-cells. This process entails the targeted alteration of genes that govern cellular differentiation and the production of insulin.
Immune System Modulation: Through the utilization of CRISPR for the modification of immune system genes, it becomes feasible to mitigate the autoimmune assault on β-cells. This may involve the generation of cells that elicit a diminished immune response or the enhancement of the immune system's tolerance towards these cells.
Advances in Cell Therapy: Preliminary investigations and clinical trials, such as those involving VCTX211, indicate significant potential for CRISPR-enhanced cellular therapies. These studies concentrate on the development of β-cells capable of exhibiting improved survivability post-transplantation and evading immune-mediated rejection.
Enhanced Gene Editing: CRISPR has facilitated the attainment of precise genetic modifications that augment the functionality and resilience of therapeutic cells. This advancement encompasses the enhancement of their resistance to immune assaults and the optimization of their insulin production efficacy.
Conclusion: CRISPR technology proffers a revolutionary strategy for the treatment of T1D by enabling meticulous genetic alterations. Although challenges persist, including the imperative to ensure safety and navigate ethical considerations, ongoing research continues to demonstrate promise in the quest to devise effective therapies that target the root causes of T1D. Subsequent investigations and clinical trials will be paramount in fully actualizing the potential of CRISPR in combatting this intricate disease.