DOI: https://doi.org/10.63345/ijrmp.v10.i11.1
Ritika Marak
Independent Researcher
Laitumkhrah, Meghalaya, India
Abstract
Messenger RNA (mRNA)-based therapeutics have emerged as a groundbreaking platform for the treatment of a wide array of genetic disorders. Unlike traditional gene therapy, which often relies on DNA delivery, mRNA therapeutics offer a transient, controllable, and non-integrative approach that minimizes risks associated with genomic alterations. This manuscript reviews the current state of mRNA therapeutic efficacy in addressing genetic disorders by providing an overview of their design, delivery systems, and mechanisms of action. Focusing on literature up to 2020, we explore the evolution of mRNA synthesis techniques, modifications to enhance stability and translation, and advances in nanoparticle and viral vector delivery. Our methodology combines a systematic literature review with an analysis of preclinical and clinical data from various studies. The findings indicate that mRNA-based therapies hold significant promise due to their ability to induce rapid protein expression and stimulate the innate immune response when required. However, challenges remain in optimizing delivery, reducing immunogenicity, and ensuring precise dosing. We conclude that while mRNA therapeutics have revolutionized vaccine development, their full potential in treating a broader spectrum of genetic disorders depends on addressing current limitations through further research and technological innovation. This review provides critical insights and recommendations for future studies aimed at harnessing the full capabilities of mRNA therapeutics in personalized medicine.
Keywords
mRNA therapeutics; genetic disorders; gene therapy; molecular medicine; nanoparticle delivery; personalized medicine
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