Scientists believe that the first gene therapy treatment for kidney disease could potentially spare children from a lifetime of dialysis or transplant procedures.
A breakthrough has been made in the treatment of nephrotic syndrome, a condition usually diagnosed in children under the age of five, which occurs when the filtering units of the kidneys are damaged. This damage leads to the leakage of a large amount of protein from the bloodstream into the urine.
Steroids can help manage the symptoms of nephrotic syndrome, but approximately 10 percent of children do not respond to them. These children are at risk of kidney failure and may require dialysis or a transplant within a few years.
In these patients, it is believed that a faulty gene responsible for producing a protein called podocin is the cause of the condition. Podocin is essential for the proper functioning of podocytes, which play a critical role in the kidney filtration system.
Researchers have found that a single dose of gene therapy in mice could replace the faulty gene. Professor Moin Saleem from the University of Bristol, who led the study, stated, “We are hopeful that this treatment could be a cure. Since podocytes are present for life, if we can modify their gene expression at the early stages of the disease, we should be able to prevent its progression. There is a window of opportunity before irreversible kidney damage occurs, and we aim to intervene with gene therapy to avoid the need for dialysis or transplantation.”
The gene therapy technique used a harmless virus to deliver genetic information into kidney cells. This replaced the faulty gene in the podocytes.
While gene therapies have been utilized for treating other conditions, this would be the first gene therapy for kidney disease. Dr. Aisling McMahon from Kidney Research UK commented, “If successful, this could rescue individuals from the burdensome dialysis treatment or the need for a transplant.”