Can we use gene therapy to treat childhood bladder disease and protect the kidneys?

27 March 2025

Dr Neil Roberts from the University of Manchester has received a PhD Studentship grant award of £85,000 to build upon previous Kidney Research UK-supported work into the potential for gene therapy to treat genetic childhood bladder disease, with the aim of protecting the kidneys.

Dr Neil Roberts

“We have already shown that gene therapy can be successful in laboratory models of certain inherited bladder disorders that cause kidney failure. The latest funding from Kidney Research UK will allow us to take the next steps to bring gene therapy closer to patients by looking at how best to use these treatments in human tissue, while also looking at whether this approach can work in other related disorders.” Dr Neil Roberts

The problem

Children who are born with incompletely or poorly formed bladders have to live with a range of symptoms and are often at particular risk of kidney damage owing to frequent infections. Children living with these conditions may be offered a range of supportive procedures, but there is no cure.

In a previous fellowship with Kidney Research UK, Neil and the team from Manchester – for the first time – showed the potential of gene therapy to treat a rare, inherited disorder called urofacial syndrome. In this syndrome the nerves and muscles in the bladder do not work together well, which can lead to multiple severe urine infections, followed by kidney damage. UFS also impacts the nerves in the face, particularly in relation to smiling.

What is gene therapy

Gene therapy is a technique where a faulty gene is replaced or altered to treat or prevent disease. Conditions that result from changes to a single gene are particularly good candidates for gene therapy. Gene therapy offers hope of new and effective treatments for certain inherited medical conditions that are currently not curable.

The solution

Building on this initial work in urofacial syndrome, Neil will now look to bring this therapy closer to the clinic. In collaboration with Professor Adrian Woolf in Manchester and Professor David Long from University College London, they will investigate how best to deliver gene therapy using human tissue samples.

In addition, the team will investigate this approach in other inherited urinary tract and kidney conditions, forming a treatment avenue for other related disorders.

Together with patient groups they aim to understand current treatments, unmet needs and attitudes towards new therapies.

An image of a model bladder which is green an red in colour. It shows the ureter tubes at the top.

A model bladder with muscle fibres in green and nerves in red. The main part of the bladder (the body) is on the left and the outflow (or urethra) is facing down to the right. The nerves start in the region between the body and the outflow (the neck) and project from there into the muscle of the body and outflow. The bunny ears at the top are ureters. Image taken MSc student, Andrew Thom.

What could this mean for kidney patients?

This research shows the potential for using gene therapy as a treatment for childhood bladder conditions and in turn, protecting the kidneys. This offers hope for families living with genetic causes of kidney diseases.