RNA-Based Therapies in Kidney Diseases.

Abstract

Kidney diseases are major global health challenges, affecting over 750 million people worldwide. Despite significant efforts, effective treatment strategies are still insufficient. In recent years, RNA therapeutics have made substantial progress, and an increasing number of nucleic acid-based therapies have been approved, showing potential for treating various diseases (including kidney diseases). These therapies can target proteins, transcripts, and genes that were previously considered "undruggable", allowing for the regulation of their expression and the expansion of therapeutic targets. Among RNA therapeutics, mRNA-based therapies are particularly promising because they can rapidly express therapeutic proteins, along with their design flexibility and potential to target previously inaccessible disease mechanisms. This review discussed various RNA-based strategies for developing new treatments, including antisense and RNA interference technologies, mRNA-based approaches, and CRISPR-Cas-mediated genome editing. Additionally, we highlighted the opportunities and challenges associated with the widespread application of these therapies in kidney disease treatment.