International Journal of Molecular Sciences (Dec 2023)

Applications of Genome-Editing Technologies for Type 1 Diabetes

  • Rana El Nahas,
  • Mohammad Ameen Al-Aghbar,
  • Laura Herrero,
  • Nicholas van Panhuys,
  • Meritxell Espino-Guarch

DOI
https://doi.org/10.3390/ijms25010344
Journal volume & issue
Vol. 25, no. 1
p. 344

Abstract

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Type 1 diabetes (T1D) is a chronic autoimmune disease characterized by the destruction of insulin-producing pancreatic β-cells by the immune system. Although conventional therapeutic modalities, such as insulin injection, remain a mainstay, recent years have witnessed the emergence of novel treatment approaches encompassing immunomodulatory therapies, such as stem cell and β-cell transplantation, along with revolutionary gene-editing techniques. Notably, recent research endeavors have enabled the reshaping of the T-cell repertoire, leading to the prevention of T1D development. Furthermore, CRISPR–Cas9 technology has demonstrated remarkable potential in targeting endogenous gene activation, ushering in a promising avenue for the precise guidance of mesenchymal stem cells (MSCs) toward differentiation into insulin-producing cells. This innovative approach holds substantial promise for the treatment of T1D. In this review, we focus on studies that have developed T1D models and treatments using gene-editing systems.

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