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Thanks to a $5.8 million grant, BWH researchers are expanding their efforts to learn about and treat Polycystic Kidney Disease (PKD), an incurable genetic disorder that affects 12 million people globally every year.
The National Institutes of Health (NIH) awarded the five-year grant to researchers last fall to establish the Harvard Interdisciplinary Center of Polycystic Kidney Disease Research, based in the Renal Division of BWH, Children’s Hospital and Harvard Medical School. This center is one of only three research centers in the country supported by the NIH to study PKD.
“The grant we received will allow us to perform critical experiments to better understand PKD,” Jing Zhou, MD, PhD, director of the center, said. “Fundamentally, this disease is not understood, and further research is necessary to study its mechanisms. We will use this basis to develop effective treatments.”
PKD is the most common genetic life-threatening disease caused by the mutation of a single gene; each child of a parent with PKD has a 50 percent chance of inheriting it. PKD causes cysts to form on the kidneys. As the cysts grow and multiply, the kidneys expand, ultimately causing end-stage renal disease. Cysts also form in other organs such as the liver and pancreas. Vascular complications such as intracranial aneurysms cause high mortality.
Dialysis and transplantation are the only forms of known treatment, but researchers in the center hope to change that. During the last 13 years, BWH researchers in Zhou’s lab have made a number of important discoveries in the understanding of this disease. These include discoveries of the functions of the disease proteins, the creation of the first animal model of PKD to test therapies and other molecular mechanisms of the disease.
With the NIH grant, Zhou recruited researchers Bradley Denker, MD, Jordan Kreidberg, PhD, Peter Vassilev, MD, Joseph Bonventre, MD, PhD, Ben Humphreys, MD, PhD, and Jagesh Shah, PhD. to pursue a solid understanding of the disease and establish treatments to control and cure it.
“We are very excited about this opportunity,” Zhou said. “It gives our dedicated researchers the chance to test our hypotheses, which could potentially result in groundbreaking discoveries for PKD.”