Researchers within the Duke Department of Surgery have published a study in Nature Biomedical Engineering that describes the discovery of adeno-associated virus (AAV) vectors specifically designed for kidney gene delivery.
The study was led by Aravind Asokan, PhD, Professor in Surgery in the Division of Surgical Sciences, and Andrew Barbas, MD, Associate Professor of Surgery in the Division of Abdominal Transplant Surgery.
The team’s findings present a clinically relevant gene delivery platform with promising applications for kidney transplantation, through ex vivo gene therapies, and chronic kidney disease, which affects more than 10% of the global population.
Kidney gene delivery has been historically challenging due to anatomical and physiological complexities of the kidney. Additionally, differences between animal models and humans have made the translation of preclinical research difficult.
To combat these challenges, researchers utilized directed evolution of AAV, an approach that has already shown tremendous promise in enabling gene therapy for the brain and muscle, for the first time in the context of the kidney.
“These new AAV vectors…open avenues for developing therapeutic modalities on gene therapy for kidney disease…as well as approaches for ex vivo genetic modification of kidneys to improve transplant outcomes,” says Dr. Asokan in a Nature Reviews Nephrology research highlight.
These potential applications presented by the study reflect the value of collaboration between basic and clinical scientists.
The study was made possible due to funding from the National Institutes of Health granted to Dr. Asokan, Dr. Barbas, and study collaborators from the Vanderbilt University Department of Cell and Developmental Biology.