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CIRM issues grant for Canavan disease
May 2016
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DURATE, Calif.—The California Institute for Regenerative Medicine (CIRM) recently made a $7.38-million grant to City of Hope to support a research team led by Dr. Yanhong Shi, director of the Division of Stem Cell Biology Research, in their efforts to develop a novel treatment for Canavan disease, a rare, fatal neurological disease that affects infants and usually causes death before age 10. Previous work by Shi on Canavan disease, which was also funded by a CIRM grant, took stem cells from Canavan disease patients and corrected the genetic defect that causes the disorder. The corrected cells were found to reduce disease impact when transplanted into mice with Canavan disease. Moving forward, research will be focused on developing a process that leads to an in-human clinical trial including stem cell transplantation in patients using their own reconfigured stem cells.
 
“Our hope is that one day, parents of children with this devastating disease will be able to watch their children grow up,” Shi said.

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