Gene Therapy Method Developed to Fight Chronic Kidney Disease
Chronic kidney disease affects over 30 million Americans, most of whom are unclear they are affected until later, much more dire stages. Current treatments are
limited in the later stages particularly: kidney transplants or dialysis.
“Chronic kidney disease is an enormous and growing problem,” said senior author Benjamin D. Humphreys, MD, PhD, director of the Division of Nephrology at Washington University. “Unfortunately, over the years, we haven’t developed more effective drugs for the condition, and this reality is leading us to explore gene therapy. Part of the reason there have been so few advances in kidney disease treatment is because the kidney is complex, and we don’t fully understand the disease process”
The Washington University School of Medicine in St. Louis has discovered genetic material in mice can be administered to damaged cells in the kidneys, a vital step toward developing gene therapy to treat chronic kidney disease.
The researchers assessed six adeno-associated viruses (AAV), natural and synthetic, in mice and in stem-cell-derived human kidney organoids. Anc80, a synthetic virus, proved successful in mice for reaching two kinds of cells that are main contributors of chronic kidney disease. The same virus was later used as gene therapy to heal scarring in mice, which also proved successful.
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