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Gene Therapy Shows Promise for Rare Children's Brain Disorder
Date:12/19/2012

sm of [brain] cells will be the cure," Leone said.

More information

Learn more about Canavan disease from the Canavan Research Foundation.

SOURCES: Paola Leone, Ph.D., associate professor, cell biology, University of Medicine and Dentistry of New Jersey, Stratford, N.J.; Morris Baslow, Ph.D., staff scientist, Nathan S. Kline Institute for Psychiatric Research, Orangeburg, N.Y.; Maria L. Escolar, M.D., director, program for the study of neurodevelopment in rare disorders, Children's Hospital of Pittsburgh; Dec. 19, 2012, Science Translational Medicine, online


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