CRISPR-Cas9 Gene Editing for Restoring Pancreatic Beta Cell Function in Diabetes
Taliikwa Nicholas Ceaser
Department of Pharmacognosy Kampala International University Uganda
Email:ceaser.taliikwa@studwc.kiu.ac.ug
ABSTRACT
Diabetes mellitus remained a leading metabolic disorder characterized by impaired insulin production and secretion due to β-cell dysfunction or loss. Conventional pharmacotherapy alleviates hyperglycemia but does not restore endogenous β-cell mass. The discovery of clustered regularly interspaced short palindromic repeats (CRISPR) and associated protein 9 (Cas9) has transformed genetic engineering, providing an unprecedented platform for targeted gene correction, activation, or repression in β-cell biology. This review critically examined current advances in CRISPR-Cas9–mediated gene editing as a strategy to restore pancreatic β-cell function in both Type 1 and Type 2 diabetes. Literature from PubMed, Scopus, and Web of Science (2013–2025) was reviewed using keywords CRISPR-Cas9, beta-cell regeneration, Type 1 diabetes, Type 2 diabetes, and gene therapy. Only peer-reviewed experimental and translational studies were included. CRISPR-Cas9 enabled precise correction of monogenic defects (e.g., INS, PDX1, GLIS3), modulation of β-cell differentiation pathways, and reprogramming of non-β pancreatic cells into insulin-producing phenotypes. In autoimmune diabetes, CRISPR-based deletion of HLA molecules or induction of immune tolerance protected β-cells from cytotoxic destruction. In metabolic diabetes, editing genes regulating endoplasmic reticulum stress, oxidative balance, and insulin exocytosis enhances β-cell resilience. However, off-target activity, low editing efficiency, and delivery barriers constrain clinical translation. CRISPR-Cas9 offered a mechanistically rational, potentially curative approach for β-cell restoration in diabetes, though rigorous optimization of delivery vectors, safety profiles, and ethical frameworks remains essential.
Keywords: CRISPR-Cas9, Pancreatic β-cell regeneration, Gene therapy, Type 1 diabetes, Type 2 diabetes.
CITE AS: Taliikwa Nicholas Ceaser (2026). CRISPR-Cas9 Gene Editing for Restoring Pancreatic Beta Cell Function in Diabetes. IAA Journal of Scientific Research 13(1):18-22.