Gene Therapy Method Developed to Target Damaged Kidney Cells
Source: Washington University in St. Louis
Summary: Researchers has shown, in mice, that genetic material can be delivered to damaged cells in the kidneys, a key step toward developing gene therapy to treat chronic kidney disease.
The potentially fatal condition affects 30 million Americans, most of whom don’t realize they have chronic kidney disease. No cure exists, and current treatments for end-stage disease mostly are limited to dialysis and kidney transplant. However, the researchers said gene therapy could provide a way to deliver genes that slow or reverse cell damage that leads to chronic kidney disease. Diabetes, hypertension and other conditions cause chronic kidney disease, which occurs when damaged kidneys cannot effectively filter waste and excess fluids from the body. Researchers from the Washington University in St. Louis has shown, in mice, that genetic material can be delivered to damaged cells in the kidneys, a key step toward developing gene therapy to treat chronic kidney disease. The study findings were published in the Journal of the American Society of Nephrology.
The team focused on whether adeno-associated virus (AAV), a relative of the virus that causes the common cold, could deliver genetic material to targeted kidney cells. Until now, no such virus has been capable of delivering genetic material to the kidney, and the new research provides a proof of concept for this approach. The researchers evaluated six AAV viruses, both natural and synthetic, in mice and in stem-cell-derived human kidney organoids. A synthetic virus, Anc80, created by one of the researchers proved successful in reaching two types of cells that contribute to chronic kidney disease; these cells secrete proteins that gum up the organ and cause irreversible damage. The researchers also showed that the genetic material carried by Anc80 was transferred successfully to the targeted kidney cells. That same virus also was used by the researchers in gene therapy strategies to treat mice with kidney scarring.
Prof. Humphreys said, “The interesting thing about the adeno-associated viruses is that they persist in the body for many months, potentially giving a therapeutic gene a chance to do its work, Chronic kidney disease is a slowly progressive disease so that is an advantage. After many more years of research, we could envision that patients would need injections maybe twice a year as opposed to every week, like with chemo.”
More Information: Yoichiro Ikeda et al, “Efficient Gene Transfer to Kidney Mesenchymal Cells Using a Synthetic Adeno-Associated Viral Vector,” Journal of the American Society of Nephrology (2018). DOI: 10.2215/ASN.2018004026