Preclinical gene therapy for genetic urinary bladder disease

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In this project we will test cures for a devastating bladder disease. This disease runs in families and makes children and adults unable to fully empty their bladders. Instead, urine builds up in the bladder and becomes infected, causing kidney failure. At present there is no cure for this disease and affected people need to put a catheter into their bladder several times a day to drain urine.Our Manchester research team have identified two faulty genes in people who have this disease. We discovered that these genes control how nerves grow into the bladder. These same nerves then control how our bladders fill up and store urine, and also control how our bladders change shape to let us pass water. We also study mice with the same genetic condition. Like people with the disease, the mice have trouble emptying their bladders. In this project we will use 'gene therapy' to cure these mice that have genetic urinary bladder disease. The therapy is given to mice just after they are born to give the best chance of treating the disease before it has done too much damage.The successful outcome of our project will be a first step towards curing people with severe bladder disease beginning in childhood. This would mean that these people no longer have life-long incontinence of urine.In future, this new type of treatment could be used in other life-threatening inherited genetic diseases that affect the kidney and urinary bladder. Such diseases are important causes of kidney failure in children and young adults and yet they currently have no definitive cures.