Understanding the causes of childhood kidney failure and delivering therapies to treat them
With the help of our funding, Dr Jennifer Chandler from the University College London Great Ormond Street Institute of Child Health will investigate the causes of childhood kidney failure and will test a novel method of delivering genetic therapies to the kidney.
Gene faults can lead to kidney failure
Up to two in 100,000 children per year worldwide are diagnosed with diseases that they are born with, that cause damage to their glomeruli- the tiny filtering units of the kidney. There are currently no treatments available to slow the decline to kidney failure in these children, leaving dialysis or transplant as their only options.
Most of these diseases occur because of faults in the genes found in special cells called podocytes, which form part of the glomeruli and help to prevent proteins and other large molecules from leaking out of the blood. However, we don’t fully understand how these gene faults lead to podocyte damage in these children.
Finding the causes of childhood kidney failure
Jennifer will carry out detailed genetic analysis in animal models that have two of the most common gene faults found in children with this type of kidney damage, in order to understand the mechanisms that cause early disease. This should uncover promising therapeutic targets to treat the early damage of the glomeruli in these children.
Jennifer will then use a new approach to deliver genetic therapies directly and specifically to the podocytes to restore the levels of these promising therapeutic targets back to normal.
What does this mean for patients?
This project has the potential to both identify new treatment targets for preventing damage to the glomeruli and provide an efficient method to deliver treatments specifically to the cells that are damaged, offering hope that children with this type of kidney damage may not progress to kidney failure.
Jennifer said: “I would like to say a huge thank you to Kidney Research UK for this fellowship funding! Not only does their support mean I can focus on some vital research questions to improve our treatment of childhood renal failure, but it also means I can start on my independent journey in the field of kidney research. For early career researchers like me, this is a really important step to begin to make your mark and in doing so, I hope to make a difference to renal medicine in the near future!”
Jennifer’s work is funded by an Intermediate Training Fellowship from Kidney Research UK for £206,220
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