Advances & challenges in developing gene therapies for rare kidney diseases

Gene therapy is an enticing therapeutic option for diseases lacking an effective long-term treatment, including not only monogenic diseases, but also complex disorders such as cancer. A number of clinical trials have shown evidence of improved efficacy and safety, mainly due to advances in vector design and delivery systems. Despite the progress made in the field, one of the main challenges for gene therapy is the ability to target specific organs and even specific cell types within an organ. With its complex architecture and variety of specialized cell types, the kidney epitomizes the difficulties encountered in developing therapeutic gene delivery strategies for highly differentiated tissues. In this review, we give an overview of the anatomical aspects that make the kidney a challenging target, explore different delivery routes and discuss attempts made to deliver exogenous DNA to renal cells and to treat renal diseases using non-viral and viral approaches. Although evidence in animal models indicates that gene therapy for renal diseases is possible, its applicability to human diseases requires further research to optimize targeting and vector delivery.

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