Applications of Gene Therapy Strategies for Podoycte Function and CKD

Podocyte injury is a significant factor in the pathogenesis of chronic kidney disease, which has become increasingly abundant on a global scale. Pathological insults - including genetic mutations and oxidative stress - can disrupt the integrity of podocytes, specialized epithelial cells that are components in the glomerular filtration barrier. Potential therapeutic strategies to target podocyte dysfunction involve the application of gene therapies as well as a greater understanding of the mechanisms of podocyte injury. Showing promise in recent clinical studies, gene therapies rely on AAV vectors to deliver the treatment to the correct target with high efficacy. In addition, with the rise of FDA approvals for gene therapy treatment in humans, therapies such as PS-001 offer promising results for treating genetic kidney disorders. Beyond the therapy itself, new advances in delivery platforms can aid in minimizing systemic toxicity. However, the limited regenerative capabilities of podocytes and inadvertent side effects must still be addressed in both the creation and delivery of new treatments.