CRISPR-Based Gene Editing Approaches for Monogenic Diabetes Therapy and Precision Medicine

Namirimu Sandrah

Department of Pharmacology and Toxicology Kampala International University Uganda

Email: sandrahnamirimu@studwc.kiu.ac.ug

ABSTRACT

Monogenic diabetes represents approximately 1-5% of all diabetes cases globally, with mutations in over 40 genes causing various forms of maturity onset diabetes of the young (MODY) and neonatal diabetes mellitus. The emergence of clustered regularly interspaced short palindromic repeats (CRISPR) technology has revolutionized therapeutic approaches for genetic disorders, offering unprecedented precision in correcting disease-causing mutations. This review examined the current state of CRISPR based gene editing applications for monogenic diabetes, analyzing therapeutic strategies, molecular mechanisms, and clinical translation challenges. A comprehensive literature search was conducted using PubMed and Web of Science databases from 2012-2025, focusing on peer reviewed articles describing CRISPR applications in monogenic diabetes models and clinical studies. Recent advances demonstrated successful correction of pathogenic variants in HNF1A, HNF4A, INS, and KCNJ11 genes using base editing and prime editing technologies, with correction efficiencies ranging from 15-85% in cellular models. Clinical applications showed promise for treating MODY subtypes, with patient derived induced pluripotent stem cells (iPSCs) serving as valuable platforms for personalized therapy development. Current limitations include delivery challenges, off target effects, and regulatory considerations for germline editing. CRISPR based approaches represent a paradigm shift toward precision medicine for monogenic diabetes, with base editing and prime editing emerging as safer alternatives to traditional nuclease-based systems for therapeutic applications.

Keywords: CRISPR gene editing, Monogenic diabetes, Precision medicine, Base editing, Prime editing

CITE AS: Namirimu Sandrah (2025). CRISPR-Based Gene Editing Approaches for Monogenic Diabetes Therapy and Precision Medicine. IDOSR JOURNAL OF APPLIED SCIENCES 10(2):53-58, 2025.  https://doi.org/10.59298/IDOSRJAS/2025/102.5358