Effectiveness of RNA-Based Genetic Therapy in Treating Rare Diseases of a Genetic Nature

Abstract

RNA-based genetic therapies have emerged as a promising approach in the treatment of rare genetic diseases, offering a novel alternative to traditional therapeutic strategies. This study reviews the current literature on the effectiveness of RNA-based therapies in treating genetic disorders, focusing on advancements in mRNA, siRNA, and antisense oligonucleotides (ASOs) therapies. A qualitative, literature-based methodology was employed to analyze recent research articles, clinical trials, and case studies from the past five years. Key findings highlight the potential of RNA-based therapies to target the root causes of genetic diseases at the molecular level, such as correcting mutations, silencing defective genes, or replacing missing proteins. RNA therapies have shown particular promise in treating rare diseases like Duchenne muscular dystrophy, spinal muscular atrophy, and certain forms of inherited retinal diseases, with several therapies already reaching clinical approval. However, challenges such as delivery mechanisms, immune response, and long-term efficacy remain significant barriers to widespread adoption. This review emphasizes the need for further research to refine RNA delivery systems, assess long-term safety, and optimize therapeutic outcomes. The study concludes by suggesting that RNA-based therapies could revolutionize the treatment of rare genetic diseases, provided these challenges are addressed through continued innovation and clinical validation.