Gene therapy may help treat chronic kidney disease

Scientists have found that administering gene therapy may reverse damage in kidney cells, suggesting a potential treatment for chronic kidney disease characterised by gradual loss of its functions.

The research showed that whether adeno-associated virus (AAV) -- a relative of the virus that causes the common cold -- could deliver genetic material to damaged cells in the kidneys.

They explained that diabetes, hypertension and other conditions cause chronic kidney disease, which occurs when damaged kidneys cannot effectively filter waste and excess fluids from the body.

"Chronic kidney disease is an enormous and growing problem. Unfortunately, over the years, we have not developed more effective drugs for the condition, and this reality is leading us to explore gene therapy," said Benjamin D. Humphreys from the Division of Nephrology at Washington University in the US.

In the study, published in Journal of the American Society of Nephrology, the team evaluated six AAV viruses, both natural and synthetic, in mice and in stem-cell-derived human kidney organoids.

A synthetic virus, Anc80, created by the researchers proved successful in reaching two types of cells that contribute to chronic kidney disease by secreting proteins that gum up the organ and cause irreversible damage.

The results showed that the genetic material carried by Anc80 was transferred successfully to the targeted kidney cells and the same virus was also used in gene therapy strategies to treat mice with kidney scarring.

"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," Humphreys explained.

"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," Humphreys added.