CRISPR-Cas9: A Future in Treating Type 1 Diabetes - A Review
DOI:
https://doi.org/10.58445/rars.683Keywords:
CRISPR-Cas9, Type 1 Diabetes, TreatmentAbstract
Type 1 Diabetes is a very prevalent disease in today’s population; approximately 8.4 million people have it. Symptoms of Type 1 Diabetes include taxed breathing, vomiting, weight loss, and extreme fatigue (American Diabetes Association [ADA], 2023). Currently, there is no cure for Type 1 Diabetes, even though it is a very prevalent disease. Gene editing is a new technology that could provide more permanent solutions to T1D ailments. The CRISPR-Cas9 technology has enabled researchers to use gene-editing technology to begin to develop treatments and possible cures for Type 1 Diabetes. CRISPR-Cas9 is a gene-editing mechanism that can be used to modify DNA, providing the possibility to cure or prevent genetic diseases. Although gene editing technology is new, researchers are testing the use of CRISPR-Cas9 technologies to treat Type 1 Diabetes. CRISPR Therapeutics and ViaCyte, Inc. announced the beginning of the first clinical trial in which genetically edited cells would be used to treat Type 1 Diabetes. Together, the companies are developing the CRISPR-edited VCTX210 stem cell therapy. When discussing genetic editing as a cure for Type 1 Diabetes, there are many safety and ethical considerations that must be taken into account: the principles of beneficence and non-maleficence, the accessibility of the treatment, the long-term effects and unintended consequences of such treatments, and patient autonomy. This paper discusses the genetic causes of Type 1 Diabetes, the process of CRISPR-Cas9 gene-editing, current steps being taken to cure Type 1 Diabetes using CRISPR-Cas9, and the safety and ethical considerations of using gene editing technology.
References
American Diabetes Association. (2023). Understanding Type 1 Diabetes. American Diabetes Association. https://diabetes.org/about-diabetes/type-1
Bertrams J. (1984). The HLA association of insulin-dependent (type I) diabetes mellitus. Behring Institute Mitteilungen, (75), 89–99.
Bio-Rad. (n.d.). CRISPR-Cas Gene Editing Teaching Resources. Bio-Rad. https://www.bio-rad.com/en-us/applications-technologies/crispr-cas-gene-editing-teaching-resources?ID=Q58I0DWDLBV5
Cohrt, K. (2021). First CRISPR Therapy for Type 1 Diabetes Set to Enter Clinical Trial. CRISPR Medicine News. https://crisprmedicinenews.com/news/first-crispr-therapy-for-type-1-diabetes-set-to-enter-clinical-trial/
CRISPR Therapeutics. (2021). CRISPR Therapeutics and ViaCyte, Inc. to Start Clinical Trial of the First Gene-Edited Cell Replacement Therapy for Treatment of Type 1 Diabetes. CRISPR Therapeutics. https://crisprtx.com/about-us/press-releases-and-presentations/crispr-therapeutics-and-viacyte-inc-to-start-clinical-trial-of-the-first-gene-edited-cell-replacement-therapy-for-treatment-of-type-1-diabetes
Crux, N. B., & Elahi, S. (2017). Human Leukocyte Antigen (HLA) and Immune Regulation: How Do Classical and Non-Classical HLA Alleles Modulate Immune Response to Human Immunodeficiency Virus and Hepatitis C Virus Infections?. Frontiers in immunology, 8, 832. https://doi.org/10.3389/fimmu.2017.00832
Cruz-Tapias, P., Castiblanco, J., Anaya, J.M. Major histocompatibility complex: Antigen processing and presentation. In: Anaya JM, Shoenfeld Y, Rojas-Villarraga A, et al., editors. Autoimmunity: From Bench to Bedside [Internet]. Bogota (Colombia): El Rosario University Press; 2013 Jul 18. Chapter 10. Available from: https://www.ncbi.nlm.nih.gov/books/NBK459467/
GenEdge (2022). CRISPR Therapeutics, Sweet Spot: ViaCyte Dose First Patient with Cell Therapy for Type 1 Diabetes. Gen Genetic Engineering and Biotechnology News. https://www.genengnews.com/gen-edge/sweet-spot-crispr-therapeutics-viacyte-dose-first-patient-with-cell-therapy-for-type-1-diabetes/#:~:text=type%201%20diabetes.-,VCTX210%20is%20designed%20to%20engineer%20ViaCyte's%20CyT49%20pluripotent%20human%20stem,the%20need%20for%20chronic%20immunosuppressants.
Gostimskaya I. (2022). CRISPR-Cas9: A History of Its Discovery and Ethical Considerations of Its Use in Genome Editing. Biochemistry. Biokhimiia, 87(8), 777–788. https://doi.org/10.1134/S0006297922080090
Jiang, F., Taylor, D. W., Chen, J. S., Kornfeld, J. E., Zhou, K., Thompson, A. J., Nogales, E., & Doudna, J. A. (2016). Structures of a CRISPR-Cas9 R-loop complex primed for DNA cleavage. Science (New York, N.Y.), 351(6275), 867–871. https://doi.org/10.1126/science.aad8282
Jordanova, E. S., Philippo, K., Giphart, M. J., Schuuring, E., & Kluin, P. M. (2003). Mutations in the HLA class II genes leading to loss of expression of HLA-DR and HLA-DQ in diffuse large B-cell lymphoma. Immunogenetics, 55(4), 203–209. https://doi.org/10.1007/s00251-003-0563-z
Mobasseri, M., Shirmohammadi, M., Amiri, T., Vahed, N., Hosseini Fard, H., & Ghojazadeh, M. (2020). Prevalence and incidence of type 1 diabetes in the world: a systematic review and meta-analysis. Health promotion perspectives, 10(2), 98–115. https://doi.org/10.34172/hpp.2020.18
National Human Genome Research Institute. (2017). What are the Ethical Concerns of Genome Editing?. National Human Genome Research Institute, National Institute of Health. https://www.genome.gov/about-genomics/policy-issues/Genome-Editing/ethical-concerns
Noble, J. A., & Valdes, A. M. (2011). Genetics of the HLA region in the prediction of type 1 diabetes. Current diabetes reports, 11(6), 533–542. https://doi.org/10.1007/s11892-011-0223-x
Nordquist H, Jamil RT. Biochemistry, HLA Antigens. [Updated 2023 Apr 24]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2023 Jan-. Available from: https://www.ncbi.nlm.nih.gov/books/NBK546662/
Steck, A. K., & Rewers, M. J. (2011). Genetics of type 1 diabetes. Clinical chemistry, 57(2), 176–185. https://doi.org/10.1373/clinchem.2010.148221
Sum, V. (2022). B.C. researchers launching clinical trial for first genetically engineered stem cell-based therapy for type 1 diabetes. The University of British Columbia. https://news.ubc.ca/2022/05/12/b-c-researchers-launching-clinical-trial-for-first-genetically-engineered-stem-cell-based-therapy-for-type-1-diabetes/
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