Journal Article

The Role of Gene Therapy and RNA-Based Therapeutic Strategies in Diabetes

Alayadhi, Sarah M., Alhalwachi, Zainab, Khan, Mustafa Tariq, Al-Dhaleai, Reem Emad, Butler, Alexandra E.

2025

Overview

Gene therapy and RNA (ribonucleic acid)-based therapeutic strategies have emerged as promising alternatives to conventional diabetes treatments, significantly expanding the therapeutic landscape using viral and non-viral vectors, and RNA modalities such as mRNA (messenger ribonucleic acid), siRNA (small interfering ribonucleic acid) and miRNA (micro ribonucleic acid). Recent advancements in these fields have led to notable preclinical successes and ongoing clinical trials, yet they are accompanied by debates over safety, efficacy and ethical considerations that underscore the complexity of clinical translation. This review offers a comprehensive analysis of the underlying mechanisms by which these treatments target diabetes, critically evaluating the fundamental concepts and mechanistic insights that form their basis, while highlighting current research gaps, such as the challenges in long-term stability and efficient delivery of RNA-based therapies, and potential adverse effects associated with gene therapy techniques. By synthesizing diverse perspectives and controversies, the review outlines future directions and interdisciplinary approaches aimed at overcoming existing hurdles, ultimately setting the stage for innovative, personalized diabetes management and addressing the broader clinical and regulatory implications of these emerging therapeutic strategies.

• Publisher: MDPI AG
• Subject: Animals / Diabetes / Diabetes Mellitus - genetics / Diabetes Mellitus - therapy / Diabetes therapy / Gene expression / Gene therapy / Genes / Genetic Therapy - methods / Glucose / Health aspects / Homeostasis / Humans / Hypoglycemia / Hypoglycemic agents / Immune response / Insulin resistance / Kinases / Liver / Messenger RNA / Metabolism / MicroRNA / MicroRNAs - genetics / MicroRNAs - therapeutic use / Musculoskeletal system / Nanoparticles / Phosphorylation / Ribonucleic acid / RNA / RNA, Messenger - genetics / RNA, Messenger - therapeutic use / RNA, Small Interfering - genetics / RNA, Small Interfering - therapeutic use / Tetracycline / Tetracyclines / Type 2 diabetes / Vectors (Biology)