Scientists believe the first gene therapy treatment for kidney disease could spare children a lifetime of dialysis or even a transplant.
They have made a breakthrough on nephrotic syndrome, usually diagnosed in kids under five, which occurs when the organ’s filtering units are damaged. That allows large amounts of protein that should be kept in the bloodstream to leak into the urine.
Symptoms can be managed with steroids but around 10 percent of children do not respond to them.
They are at risk of kidney failure and often need dialysis or a transplant within two to three years.
In these patients, a faulty gene that codes for a protein called podocin is thought to be the cause. Podocin is essential for the functioning of cells called podocytes which play a critical role in the kidney filtration system.
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Research in mice has found just one dose of gene therapy could replace the faulty gene. Study leader Professor Moin Saleem, of the University of Bristol, said: “We are hoping that this treatment could be curative.
You keep the same podocytes for life, so if we can change their gene expression right at the beginning of the disease, we should be able to prevent it from progressing.
“There is a reasonable window of opportunity before you get irreversible damage to the kidneys, where we would hope to be able to intervene with gene therapy and avoid the need for dialysis or transplantation.”
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The gene therapy used a virus that was incapable of causing disease to carry genetic information into kidney cells. This replaced the original faulty copy of the gene in the podocytes.
Gene therapies have been used to treat other conditions but this would be the first for kidney disease.
Dr Aisling McMahon, of Kidney Research UK, told Science Trans-lational Medicine: “If successful, this could rescue people from gruelling dialysis treatment or a transplant.”
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