Gene therapy offers promise to children battling kidney disease


Scientists are looking for better ways to help kids with kidney disease. They think gene therapy might be a good solution. Gene therapy is a new and smart way to fix the problems causing kidney diseases in children. This new study talks about how gene therapy could be excellent for kids with kidney problems. It’s like a bright light of hope for these young patients and their families.

Gene therapy can cure a kidney problem called steroid-resistant nephrotic syndrome. The research got support from Kidney Research UK, the Wellcome Trust, and Purespring Therapeutics. The study, led by Professor Moin Saleem and his team in Bristol at University of Bristol, discovered that fixing a gene that’s not working correctly could help a protein called podocin. This protein is essential for special cells in the kidney called podocytes, which help filter the blood. The research was published in a journal called Science Translational Medicine.

Nephrotic syndrome cause when the kidneys have a problem and inject too much essential protein into the urine. This situation can make parts of the body swell, like the eyes and legs. It also raises the chances of infections, blood clots, and kidney damage. Kids under five years old often get this problem.

Doctors usually use steroids to help, but some kids (about 10%) don’t get better with it. These kids might need dialysis or a kidney transplant in a few years. It happens because of a gene problem, like their body’s instruction manual.

Professor Saleem said, “We hope 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 this disease from progressing. With most kidney diseases, there is a reasonable window of opportunity, often years, 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.”

A significant discovery could help lots of kids with nephrotic syndrome. These kids might have deficient kidneys and need unique treatments like dialysis or transplants. But if this new thing works, they might not need those treatments anymore.

For this to work, scientists must ensure the new stuff they put in the body fixes the problem for a long time. They used a special adeno-associated virus (AAV) to carry the new part into the suitable cells. This virus doesn’t make sick, but it’s good at delivering important things to cells. With this method, the scientists replaced the bad gene in the kidney cells and improved stuff in lab tests. It is a big step towards helping kids with nephrotic syndrome.

Dr. Aisling McMahon, a leader at Kidney Research UK, shared that if this new method works, it could save people from having to undergo difficult dialysis or get a new kidney. It’s hopeful news for patients with nephrotic syndrome and other kidney problems. The funding from Kidney Research UK has helped move this project forward, and it could become a natural treatment option.

Researchers are using gene therapy with the AAV virus, which was already used for other illnesses. But this is the first time it’s been tried for kidney issues. They need to check how much to use and ensure it’s safe. Even so, researchers believe it might be soon that doctors can use this method to help patients. In the next few years, more studies will decide if this can treat people’s kidney problems.

Journal Reference:

  1. Wen Y. Ding, Valeryia Kuzmuk et al., Adeno-associated virus gene therapy prevents progression of kidney disease in genetic models of nephrotic syndrome. Science Translation Medicine. DOI: 10.1126/scitranslmed.abc8226.