Gene therapy hope for children with kidney disease

Researchers at the University of Bristol have made a remarkable step forward in finding a potential cure for a type of childhood kidney disease.

The research project has shown that just one dose of gene therapy targeting cells in the kidney has the potential to cure a condition known as steroid-resistant nephrotic syndrome.

The research project received early funding from charity Kidney Research UK and the Wellcome Trust and has gone on to gain further support from Purespring Therapeutics.

The findings, which were published in the journal Science Translational Medicine by the team in Bristol led by Professor Moin Saleem, from the Bristol Medical School, suggested that replacing one faulty gene that codes for a protein known as podocin could cure the condition.