Preprint / Version 1

Gene Editing with CRISPR: A Novel Approach to Type 1 Diabetes Management

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  • William Luo Mission San Jose High School

DOI:

https://doi.org/10.58445/rars.3199

Keywords:

CRISPR, type 1 diabetes, CTLA4, single nucleotide polymorphism, Akita mutation

Abstract

Type one diabetes, a genetic predisposition, affects roughly 8.4 million people around the globe. It is a chronic, autoimmune condition where the body’s immune cells respond to and attack insulin-producing cells within the pancreas. While there is currently no known treatment for type one diabetes, there have been many proposed treatments for type 1 diabetes, the most prevalent being the CRISPR-Cas9 gene editing complex. In this study, we will be exploring various genetic sequences CRISPR-Cas9 can edit to treat type 1 diabetes, and explore the potential downstream effects of either non-homologous end joining, homology directed repair, or site directed mutagenesis of the proposed sequences.

References

Asmamaw, Tadesse, et al. “CRISPR/Cas9 Gene Editing: Mechanism and Applications.” Journal of Genetic Engineering and Biotechnology, vol. 19, no. 1, 2021, pp. 1–12.

Buchbinder, Elizabeth, and Anisha Desai. “CTLA-4 and Immune Checkpoint Blockade.” Cancer Journal, vol. 24, no. 2, 2018, pp. 65–71.

Burrack, K. S., et al. “Type 1 Diabetes: From Pathogenesis to Therapy.” Frontiers in Immunology, vol. 9, 2018, article 2539.

Chen, Wei, et al. “Association of CTLA4 Gene Polymorphisms with Type 1 Diabetes in the Chinese Han Population.” Diabetes Research and Clinical Practice, vol. 104, no. 2, 2014, pp. 219–225.

Cleveland Clinic. “Type 1 Diabetes.” Cleveland Clinic, https://my.clevelandclinic.org/health/diseases/7104-type-1-diabetes.

GenScript. “CRISPR sgRNA Design Tool.” GenScript, https://www.genscript.com/tools/crispr-sgRNA-design-tool.

Gough, Stephen C. L., et al. “Genetics of Autoimmune Disease: Insights from CTLA4.” Nature Reviews Genetics, vol. 6, no. 5, 2005, pp. 387–398.

Gostimskya, Olga. “The Tragic Story of Jesse Gelsinger.” Science History Institute, https://www.sciencehistory.org/stories/jesse-gelsinger-gene-therapy.

Hossen, Md. Jahangir, et al. “CTLA-4 +49A/G Polymorphism and Type 1 Diabetes Risk: A Meta-Analysis.” PLOS ONE, vol. 10, no. 2, 2015, e0117085.

Liu, Mei, et al. “Mutations in the INS Gene and Their Role in Diabetes.” Endocrine Reviews, vol. 30, no. 4, 2009, pp. 343–358.

McGrail, David J., et al. “HLA Haplotypes and Risk of Type 1 Diabetes.” Immunogenetics, vol. 72, no. 3, 2020, pp. 189–200.

Noble, James A., et al. “HLA Class II Genotype and Risk of Type 1 Diabetes.” Diabetes, vol. 49, no. 2, 2000, pp. 312–316.

Nordquist, Niklas, et al. “The Role of HLA in Autoimmune Disease.” Autoimmunity Reviews, vol. 8, no. 7, 2009, pp. 595–600.

Ogle, Gregory D., et al. “Global Epidemiology of Type 1 Diabetes.” Diabetes Research and Clinical Practice, vol. 144, 2018, pp. 1–10.

Pathak, Varun, et al. “Challenges in Insulin Therapy for Type 1 Diabetes.” Journal of Diabetes Science and Technology, vol. 13, no. 2, 2019, pp. 276–283.

Chow, R.D., Chen, J.S., Shen, J. et al. “A web tool for the design of prime-editing guide RNAs.” Nature Biomedical Engineering 5, 190–194 (2021), https://doi.org/10.1038/s41551-020-00622-8

Pugliese, Alberto, and Maria Miceli. “INS Gene Expression and Regulation.” Diabetes Metabolism Research and Reviews, vol. 25, no. 6, 2009, pp. 507–517.

Redondo, María J., et al. “The Clinical Consequences of Heterogeneity in Type 1 Diabetes.” Diabetologia, vol. 63, no. 3, 2020, pp. 437–443.

Ron, David. “ER Stress and Beta Cell Death in Diabetes.” Cell Metabolism, vol. 9, no. 5, 2009, pp. 347–348.

Rowshanravan, Bita, et al. “CTLA-4: Mechanisms of Action in Immune Regulation.” Immunology, vol. 152, no. 3, 2017, pp. 459–472.

University of Massachusetts Medical School. “CRISPR/Cas9 Genome Editing.” UMass Medical School, https://www.umassmed.edu/zebrafish/protocols/crisprcas9-genome-editing.

Wang, J., et al. “Akita Mouse Model of Type 1 Diabetes.” Diabetes, vol. 48, no. 5, 1999, pp. 1020–1029.

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2025-10-10

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