In patients with brain disorder Joubert syndrome, gene-editing could stop kidney disease, Kidney Research UK researchers have found. Not all patients with the syndrome carry the CEP290 faulty gene, but those who have it develop kidney disease and may eventually need a transplant. In a study funded by Newcastle University, scientists used a strand of gene-edited DNA to “trick” a cell's own systems into bypassing the mutation, known as exon-skipping. It is the first time gene editing has been carried out within a kidney, as it was previously thought to be too challenging, said lead researcher, Professor John Sayer.
In patients with brain disorder Joubert syndrome, gene-editing could stop kidney disease, Newcastle University researchers have found. Not all patients with the syndrome carry the CEP290 faulty gene, but those who have it develop kidney disease and may eventually need a transplant. In a study funded by Kidney Research UK, scientists used a strand of gene-edited DNA to “trick” a cell's own systems into bypassing the mutation, known as exon-skipping. It is the first time gene editing has been carried out within a kidney, as it was previously thought to be too challenging, said lead researcher, Professor John Sayer.