Gene Therapies: Any Merit in Nephrology?

Gene therapy is an innovative medical approach that involves altering or replacing defective genetic material to treat or potentially cure genetic disorders. This technique primarily uses viral or non-viral vectors to deliver genetic material into cells, aiming to restore normal gene function. The therapy has the potential to address a wide range of diseases, including genetic, cardiovascular, and neurodegenerative disorders. In this review, the focus will be on gene therapies related to kidney diseases. Topics to be covered include the use of messenger ribonucleic acid (mRNA) therapies for conditions such as hypertension and kidney cancer, as well as targeted gene therapies using small interfering RNA (siRNA) and adeno-associated viruses to treat glomerular diseases and prevent kidney damage. The application of gene therapies in treating well-known genetic conditions, such as Alport syndrome and cystinosis, will also be discussed. Additionally, the review will explore the progress of RNA interference (RNAi) therapies in acute kidney injury (AKI) and chronic kidney disease (CKD). Finally, the challenges and risks associated with gene therapy, including immune responses, insertional mutagenesis, and the high costs of treatment, will be examined.